{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,5]],"date-time":"2026-10-05T10:22:37Z","timestamp":1791195757826,"version":"4.1.0"},"reference-count":288,"publisher":"MDPI AG","issue":"2","license":[{"start":{"date-parts":[[2021,2,19]],"date-time":"2021-02-19T00:00:00Z","timestamp":1613692800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100007052","name":"Universit\u00e0 degli Studi di Verona","doi-asserted-by":"publisher","award":["754345"],"award-info":[{"award-number":["754345"]}],"id":[{"id":"10.13039\/501100007052","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pharmaceutics"],"abstract":"<jats:p>Muscular Dystrophies (MDs) are a group of rare inherited genetic muscular pathologies encompassing a variety of clinical phenotypes, gene mutations and mechanisms of disease. MDs undergo progressive skeletal muscle degeneration causing severe health problems that lead to poor life quality, disability and premature death. There are no available therapies to counteract the causes of these diseases and conventional treatments are administered only to mitigate symptoms. Recent understanding on the pathogenetic mechanisms allowed the development of novel therapeutic strategies based on gene therapy, genome editing CRISPR\/Cas9 and drug repurposing approaches. Despite the therapeutic potential of these treatments, once the actives are administered, their instability, susceptibility to degradation and toxicity limit their applications. In this frame, the design of delivery strategies based on nanomedicines holds great promise for MD treatments. This review focuses on nanomedicine approaches able to encapsulate therapeutic agents such as small chemical molecules and oligonucleotides to target the most common MDs such as Duchenne Muscular Dystrophy and the Myotonic Dystrophies. The challenge related to in vitro and in vivo testing of nanosystems in appropriate animal models is also addressed. Finally, the most promising nanomedicine-based strategies are highlighted and a critical view in future developments of nanomedicine for neuromuscular diseases is provided.<\/jats:p>","DOI":"10.3390\/pharmaceutics13020278","type":"journal-article","created":{"date-parts":[[2021,2,19]],"date-time":"2021-02-19T09:06:10Z","timestamp":1613725570000},"page":"278","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":33,"title":["Nanomedicine for Gene Delivery and Drug Repurposing in the Treatment of Muscular Dystrophies"],"prefix":"10.3390","volume":"13","author":[{"given":"Ilaria","family":"Andreana","sequence":"first","affiliation":[{"name":"Laboratoire d\u2019Automatique, de G\u00e9nie des Proc\u00e9d\u00e9s et de G\u00e9nie Pharmaceutique, Universit\u00e9 Claude Bernard Lyon 1, CNRS UMR 5007, 43 bd 11 Novembre 1918, 69622 Villeurbanne, France"},{"name":"Department of Drug Science and Technology, University of Turin, Via P. Giuria 9, 10125 Torino, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0613-3667","authenticated-orcid":false,"given":"Mathieu","family":"Repellin","sequence":"additional","affiliation":[{"name":"Laboratoire d\u2019Automatique, de G\u00e9nie des Proc\u00e9d\u00e9s et de G\u00e9nie Pharmaceutique, Universit\u00e9 Claude Bernard Lyon 1, CNRS UMR 5007, 43 bd 11 Novembre 1918, 69622 Villeurbanne, France"},{"name":"Department of Neurosciences, Biomedicine and Movement Sciences, Anatomy and Histology Section, University of Verona, Strada Le Grazie 8, 37134 Verona, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Flavia","family":"Carton","sequence":"additional","affiliation":[{"name":"Department of Neurosciences, Biomedicine and Movement Sciences, Anatomy and Histology Section, University of Verona, Strada Le Grazie 8, 37134 Verona, Italy"},{"name":"Department of Health Sciences, University of Eastern Piedmont, Via Solaroli 17, 28100 Novara, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Kryza","sequence":"additional","affiliation":[{"name":"Laboratoire d\u2019Automatique, de G\u00e9nie des Proc\u00e9d\u00e9s et de G\u00e9nie Pharmaceutique, Universit\u00e9 Claude Bernard Lyon 1, CNRS UMR 5007, 43 bd 11 Novembre 1918, 69622 Villeurbanne, France"},{"name":"Hospices Civils de Lyon, 69437 Lyon, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7997-0410","authenticated-orcid":false,"given":"St\u00e9phanie","family":"Brian\u00e7on","sequence":"additional","affiliation":[{"name":"Laboratoire d\u2019Automatique, de G\u00e9nie des Proc\u00e9d\u00e9s et de G\u00e9nie Pharmaceutique, Universit\u00e9 Claude Bernard Lyon 1, CNRS UMR 5007, 43 bd 11 Novembre 1918, 69622 Villeurbanne, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1262-502X","authenticated-orcid":false,"given":"B\u00e9n\u00e9dicte","family":"Chazaud","sequence":"additional","affiliation":[{"name":"Institut NeuroMyoG\u00e8ne, University of Lyon, INSERM U1217, CNRS UMR 5310, 8 Avenue Rockefeller, 69008 Lyon, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"R\u00e9mi","family":"Mounier","sequence":"additional","affiliation":[{"name":"Institut NeuroMyoG\u00e8ne, University of Lyon, INSERM U1217, CNRS UMR 5310, 8 Avenue Rockefeller, 69008 Lyon, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Silvia","family":"Arpicco","sequence":"additional","affiliation":[{"name":"Department of Drug Science and Technology, University of Turin, Via P. Giuria 9, 10125 Torino, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8196-9232","authenticated-orcid":false,"given":"Manuela","family":"Malatesta","sequence":"additional","affiliation":[{"name":"Department of Neurosciences, Biomedicine and Movement Sciences, Anatomy and Histology Section, University of Verona, Strada Le Grazie 8, 37134 Verona, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8266-6604","authenticated-orcid":false,"given":"Barbara","family":"Stella","sequence":"additional","affiliation":[{"name":"Department of Drug Science and Technology, University of Turin, Via P. Giuria 9, 10125 Torino, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giovanna","family":"Lollo","sequence":"additional","affiliation":[{"name":"Laboratoire d\u2019Automatique, de G\u00e9nie des Proc\u00e9d\u00e9s et de G\u00e9nie Pharmaceutique, Universit\u00e9 Claude Bernard Lyon 1, CNRS UMR 5007, 43 bd 11 Novembre 1918, 69622 Villeurbanne, France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2021,2,19]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"1009","DOI":"10.1016\/j.ncl.2013.04.004","article-title":"Muscular dystrophies and other genetic myopathies","volume":"31","author":"Shieh","year":"2013","journal-title":"Neurol. Clin."},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1159\/000369343","article-title":"Prevalence of muscular dystrophies: A systematic literature review","volume":"43","author":"Theadom","year":"2014","journal-title":"Neuroepidemiology"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"2025","DOI":"10.1016\/S0140-6736(19)32910-1","article-title":"Muscular dystrophies","volume":"394","author":"Mercuri","year":"2019","journal-title":"Lancet Lond. Engl."},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"377","DOI":"10.1016\/j.ccm.2018.01.004","article-title":"Muscular Dystrophies","volume":"39","author":"Carter","year":"2018","journal-title":"Clin. Chest Med."},{"key":"ref_5","first-page":"1682","article-title":"Myotonic Dystrophies","volume":"25","author":"Johnson","year":"2019","journal-title":"Continuum"},{"key":"ref_6","first-page":"154","article-title":"Clinical aspects, molecular pathomechanisms and management of myotonic dystrophies","volume":"32","author":"Meola","year":"2013","journal-title":"Acta Myol."},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"1837","DOI":"10.1007\/s10072-018-3555-3","article-title":"Clinical management of Duchenne muscular dystrophy: The state of the art","volume":"39","author":"Messina","year":"2018","journal-title":"Neurol. Sci."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"5307","DOI":"10.1096\/fj.201700249R","article-title":"Phosphodiesterase 4 inhibitor and phosphodiesterase 5 inhibitor combination therapy has antifibrotic and anti-inflammatory effects in mdx mice with Duchenne muscular dystrophy","volume":"31","author":"Nio","year":"2017","journal-title":"FASEB J."},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1016\/j.lfs.2015.12.015","article-title":"Anti-fibrotic effect of pirfenidone in muscle derived-fibroblasts from Duchenne muscular dystrophy patients","volume":"145","author":"Zanotti","year":"2016","journal-title":"Life Sci."},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"451","DOI":"10.1016\/S0140-6736(17)32160-8","article-title":"Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne Muscular Dystrophy: A prospective cohort study","volume":"391","author":"McDonald","year":"2018","journal-title":"Lancet"},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"633","DOI":"10.1007\/s00431-019-03336-5","article-title":"Skeletal disproportion in glucocorticoid-treated boys with Duchenne muscular dystrophy","volume":"178","author":"Kao","year":"2019","journal-title":"Eur. J. Pediatr."},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"1040","DOI":"10.1016\/j.nmd.2012.06.354","article-title":"Bone health in boys with Duchenne muscular dystrophy on long-term daily deflazacort therapy","volume":"22","author":"Mayo","year":"2012","journal-title":"Neuromuscul. Disord."},{"key":"ref_13","doi-asserted-by":"crossref","first-page":"39","DOI":"10.1097\/MED.0000000000000456","article-title":"Growth, pubertal development, and skeletal health in boys with Duchenne muscular dystrophy","volume":"26","author":"Ward","year":"2019","journal-title":"Curr. Opin. Endocrinol. Diabetes Obes."},{"key":"ref_14","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1016\/S1474-4422(18)30024-3","article-title":"Diagnosis and management of Duchenne muscular dystrophy, part 1: Diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management","volume":"17","author":"Birnkrant","year":"2018","journal-title":"Lancet Neurol."},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"975","DOI":"10.1016\/j.ijcard.2016.08.040","article-title":"Cardiac implantable electronic devices in tracheotomized muscular dystrophy patients: Safety and risks","volume":"222","author":"Fayssoil","year":"2016","journal-title":"Int. J. Cardiol."},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"1037","DOI":"10.2340\/16501977-1874","article-title":"Decanulation of patients with severe respiratory muscle insufficiency: Efficacy of mechanical insufflation-exsufflation","volume":"46","author":"Bach","year":"2014","journal-title":"J. Rehabil. Med."},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"18551","DOI":"10.1073\/pnas.0903234106","article-title":"Pentamidine reverses the splicing defects associated with myotonic dystrophy","volume":"106","author":"Warf","year":"2009","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"1740","DOI":"10.1016\/j.drudis.2017.07.011","article-title":"Myotonic dystrophy: Candidate small molecule therapeutics","volume":"22","author":"Konieczny","year":"2017","journal-title":"Drug Discov. Today"},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"4221","DOI":"10.1073\/pnas.1117019109","article-title":"RNase H-mediated degradation of toxic RNA in myotonic dystrophy type 1","volume":"109","author":"Lee","year":"2012","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"373","DOI":"10.1038\/s41582-019-0203-3","article-title":"Therapeutic developments for Duchenne muscular dystrophy","volume":"15","author":"Verhaart","year":"2019","journal-title":"Nat. Rev. Neurol."},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"eaay6812","DOI":"10.1126\/sciadv.aay6812","article-title":"Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system","volume":"6","author":"Zhang","year":"2020","journal-title":"Sci. Adv."},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"1372","DOI":"10.1016\/j.ymthe.2019.05.021","article-title":"Genome editing of expanded CTG repeats within the human DMPK gene reduces nuclear RNA foci in the muscle of DM1 mice","volume":"27","author":"Poulard","year":"2019","journal-title":"Mol. Ther."},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"3068","DOI":"10.1021\/mp500257s","article-title":"Liposomal nitrooxy-doxorubicin: One step over caelyx in drug-resistant human cancer cells","volume":"11","author":"Pedrini","year":"2014","journal-title":"Mol. Pharm."},{"key":"ref_24","doi-asserted-by":"crossref","first-page":"1344","DOI":"10.1001\/jamaoncol.2018.2168","article-title":"Safety and efficacy of BIND-014, a docetaxel nanoparticle targeting prostate-specific membrane antigen for patients with metastatic castration-resistant prostate cancer: A phase 2 clinical trial","volume":"4","author":"Autio","year":"2018","journal-title":"JAMA Oncol."},{"key":"ref_25","doi-asserted-by":"crossref","first-page":"1007","DOI":"10.1038\/s41565-019-0567-y","article-title":"Smart cancer nanomedicine","volume":"14","author":"Sulheim","year":"2019","journal-title":"Nat. Nanotechnol."},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"78","DOI":"10.1016\/j.ejca.2018.12.007","article-title":"NAPOLI-1 phase 3 study of liposomal irinotecan in metastatic pancreatic cancer: Final overall survival analysis and characteristics of long-term survivors","volume":"108","author":"Hubner","year":"2019","journal-title":"Eur. J. Cancer"},{"key":"ref_27","doi-asserted-by":"crossref","first-page":"936","DOI":"10.1038\/s41401-020-0429-z","article-title":"Nanomedicine-Based Immunotherapy for Central Nervous System Disorders","volume":"41","author":"Hanif","year":"2020","journal-title":"Acta Pharmacol. Sin."},{"key":"ref_28","doi-asserted-by":"crossref","first-page":"4123","DOI":"10.1021\/acsnano.7b08172","article-title":"Targeted brain delivery of rabies virus glycoprotein 29-modified deferoxamine-loaded nanoparticles reverses functional deficits in Parkinsonian mice","volume":"12","author":"You","year":"2018","journal-title":"ACS Nano"},{"key":"ref_29","doi-asserted-by":"crossref","first-page":"926","DOI":"10.1007\/s12035-019-01780-w","article-title":"Gold nanoparticles treatment reverses brain damage in Alzheimer\u2019s disease model","volume":"57","author":"Arruda","year":"2020","journal-title":"Mol. Neurobiol."},{"key":"ref_30","doi-asserted-by":"crossref","first-page":"282","DOI":"10.1016\/j.nano.2018.10.001","article-title":"Overcoming challenges in treating autoimmuntity: Development of tolerogenic immune-modifying nanoparticles","volume":"18","author":"Pearson","year":"2019","journal-title":"Nanomedicine"},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"4698","DOI":"10.1039\/C9BM01025D","article-title":"Nanoparticle-delivered siRNA targeting Bruton\u2019s tyrosine kinase for rheumatoid arthritis therapy","volume":"7","author":"Zhao","year":"2019","journal-title":"Biomater. Sci."},{"key":"ref_32","doi-asserted-by":"crossref","first-page":"632","DOI":"10.1002\/art.40773","article-title":"Suppression of murine Lupus by CD4+ and CD8+ treg cells induced by T cell-targeted nanoparticles loaded with interleukin-2 and transforming growth factor \u03b2","volume":"71","author":"Horwitz","year":"2019","journal-title":"Arthritis Rheumatol."},{"key":"ref_33","doi-asserted-by":"crossref","unstructured":"Fries, C.N., Curvino, E.J., Chen, J.-L., Permar, S.R., Fouda, G.G., and Collier, J.H. (2020). Advances in nanomaterial vaccine strategies to address infectious diseases impacting global health. Nat. Nanotechnol., 1\u201314.","DOI":"10.1038\/s41565-020-0739-9"},{"key":"ref_34","unstructured":"(2020). Nanomedicine and the COVID-19 vaccines. Nat. Nanotechnol., 1."},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"594","DOI":"10.1038\/s41586-020-2814-7","article-title":"COVID-19 vaccine BNT162b1 elicits human antibody and TH1 T cell responses","volume":"586","author":"Sahin","year":"2020","journal-title":"Nature"},{"key":"ref_36","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1056\/NEJMoa2035389","article-title":"Efficacy and safety of the MRNA-1273 SARS-CoV-2 vaccine","volume":"384","author":"Baden","year":"2020","journal-title":"N. Engl. J. Med."},{"key":"ref_37","doi-asserted-by":"crossref","first-page":"12522","DOI":"10.1021\/acsnano.0c07197","article-title":"COVID-19 vaccine frontrunners and their nanotechnology design","volume":"14","author":"Chung","year":"2020","journal-title":"ACS Nano"},{"key":"ref_38","doi-asserted-by":"crossref","first-page":"396","DOI":"10.1038\/nature18300","article-title":"Systemic RNA delivery to dendritic cells exploits antiviral defence for cancer immunotherapy","volume":"534","author":"Kranz","year":"2016","journal-title":"Nature"},{"key":"ref_39","doi-asserted-by":"crossref","unstructured":"Xin, X., Kumar, V., Lin, F., Kumar, V., Bhattarai, R., Bhatt, V.R., Tan, C., and Mahato, R.I. (2020). Redox-responsive nanoplatform for codelivery of miR-519c and gemcitabine for pancreatic cancer therapy. Sci. Adv., eabd6764.","DOI":"10.1126\/sciadv.abd6764"},{"key":"ref_40","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1016\/j.biomaterials.2016.04.010","article-title":"Low dose gemcitabine-loaded lipid nanocapsules target monocytic myeloid-derived suppressor cells and potentiate cancer immunotherapy","volume":"96","author":"Sasso","year":"2016","journal-title":"Biomaterials"},{"key":"ref_41","doi-asserted-by":"crossref","first-page":"2684","DOI":"10.1200\/JCO.2017.77.6112","article-title":"CPX-351 (cytarabine and daunorubicin) liposome for injection versus conventional cytarabine plus daunorubicin in older patients with newly diagnosed secondary acute myeloid leukemia","volume":"36","author":"Lancet","year":"2018","journal-title":"J. Clin. Oncol."},{"key":"ref_42","doi-asserted-by":"crossref","unstructured":"Salvioni, L., Rizzuto, M.A., Bertolini, J.A., Pandolfi, L., Colombo, M., and Prosperi, D. (2019). Thirty years of cancer nanomedicine: Success, frustration, and hope. Cancers, 11.","DOI":"10.3390\/cancers11121855"},{"key":"ref_43","doi-asserted-by":"crossref","first-page":"318","DOI":"10.1002\/mus.22094","article-title":"Structure and function of the skeletal muscle extracellular matrix","volume":"44","author":"Gillies","year":"2011","journal-title":"Muscle Nerve"},{"key":"ref_44","doi-asserted-by":"crossref","first-page":"6089","DOI":"10.2147\/IJN.S138129","article-title":"The effects of collagen-rich extracellular matrix on the intracellular delivery of glycol chitosan nanoparticles in human lung fibroblasts","volume":"12","author":"Yhee","year":"2017","journal-title":"Int. J. Nanomed."},{"key":"ref_45","doi-asserted-by":"crossref","first-page":"160","DOI":"10.1002\/jmor.21088","article-title":"Diversity of extracellular matrix morphology in vertebrate skeletal muscle","volume":"281","author":"Sleboda","year":"2020","journal-title":"J. Morphol."},{"key":"ref_46","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1186\/s12989-017-0199-z","article-title":"Mechanistic understanding of nanoparticles\u2019 interactions with extracellular matrix: The cell and immune system","volume":"14","author":"Engin","year":"2017","journal-title":"Part. Fibre Toxicol."},{"key":"ref_47","doi-asserted-by":"crossref","first-page":"1342","DOI":"10.1016\/j.bpj.2010.06.016","article-title":"Diffusion of particles in the extracellular matrix: The effect of repulsive electrostatic interactions","volume":"99","author":"Stylianopoulos","year":"2010","journal-title":"Biophys. J."},{"key":"ref_48","doi-asserted-by":"crossref","first-page":"3633","DOI":"10.1021\/la204833h","article-title":"Bottom-up design and synthesis of limit size lipid nanoparticle systems with aqueous and triglyceride cores using millisecond microfluidic mixing","volume":"28","author":"Zhigaltsev","year":"2012","journal-title":"Langmuir"},{"key":"ref_49","doi-asserted-by":"crossref","first-page":"1700375","DOI":"10.1002\/smtd.201700375","article-title":"State-of-the-art design and rapid-mixing production techniques of lipid nanoparticles for nucleic acid delivery","volume":"2","author":"Evers","year":"2018","journal-title":"Small Methods"},{"key":"ref_50","doi-asserted-by":"crossref","first-page":"200","DOI":"10.1186\/s12967-019-1945-9","article-title":"Translational formulation of nanoparticle therapeutics from laboratory discovery to clinical scale","volume":"17","author":"Feng","year":"2019","journal-title":"J. Transl. Med."},{"key":"ref_51","doi-asserted-by":"crossref","first-page":"1327","DOI":"10.2174\/1381612820666140929095755","article-title":"Strategies for skeletal muscle targeting in drug discovery","volume":"21","author":"Ebner","year":"2015","journal-title":"Curr. Pharm. Des."},{"key":"ref_52","doi-asserted-by":"crossref","first-page":"163","DOI":"10.1017\/cjn.2015.311","article-title":"A Systematic review and meta-analysis on the epidemiology of the muscular dystrophies","volume":"43","author":"Mah","year":"2015","journal-title":"Can. J. Neurol. Sci."},{"key":"ref_53","first-page":"469","article-title":"Consensus on the diagnosis, treatment and follow-up of patients with Duchenne muscular dystrophy","volume":"34","year":"2019","journal-title":"Neurologia"},{"key":"ref_54","doi-asserted-by":"crossref","first-page":"509","DOI":"10.1016\/0092-8674(87)90504-6","article-title":"Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals","volume":"50","author":"Koenig","year":"1987","journal-title":"Cell"},{"key":"ref_55","doi-asserted-by":"crossref","first-page":"1166","DOI":"10.3892\/ijmm.2013.1498","article-title":"Novel mutation in exon 56 of the dystrophin gene in a child with Duchenne muscular dystrophy","volume":"32","author":"Zhu","year":"2013","journal-title":"Int. J. Mol. Med."},{"key":"ref_56","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1016\/S1474-4422(03)00585-4","article-title":"Dystrophin and mutations: One gene, several proteins, multiple phenotypes","volume":"2","author":"Muntoni","year":"2003","journal-title":"Lancet Neurol."},{"key":"ref_57","first-page":"498","article-title":"The molecular basis for Duchenne versus Becker muscular dystrophy: Correlation of severity with type of deletion","volume":"45","author":"Koenig","year":"1989","journal-title":"Am. J. Hum. Genet."},{"key":"ref_58","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1007\/978-1-4939-7374-3_9","article-title":"Exon skipping therapy using phosphorodiamidate morpholino oligomers in the mdx52 mouse model of Duchenne muscular dystrophy","volume":"1687","author":"Miyatake","year":"2018","journal-title":"Methods Mol. Biol."},{"key":"ref_59","doi-asserted-by":"crossref","first-page":"31","DOI":"10.1016\/j.omtn.2017.02.007","article-title":"CRISPR\/Cas9-mediated genome editing corrects dystrophin mutation in skeletal muscle stem cells in a mouse model of muscle dystrophy","volume":"7","author":"Zhu","year":"2017","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"ref_60","unstructured":"Adam, M.P., Ardinger, H.H., Pagon, R.A., Wallace, S.E., Bean, L.J., Stephens, K., and Amemiya, A. (1993\u20132020). Myotonic Dystrophy Type 1. GeneReviews\u00ae, University of Washington."},{"key":"ref_61","doi-asserted-by":"crossref","first-page":"705","DOI":"10.1016\/j.ncl.2014.04.011","article-title":"Myotonic dystrophy","volume":"32","author":"Thornton","year":"2014","journal-title":"Neurol. Clin."},{"key":"ref_62","doi-asserted-by":"crossref","first-page":"358","DOI":"10.1136\/jnnp.2008.158261","article-title":"The myotonic dystrophies: Diagnosis and management","volume":"81","author":"Turner","year":"2010","journal-title":"J. Neurol. Neurosurg. Psychiatry"},{"key":"ref_63","doi-asserted-by":"crossref","first-page":"1281","DOI":"10.1042\/BST0371281","article-title":"Pathogenic mechanisms of myotonic dystrophy","volume":"37","author":"Lee","year":"2009","journal-title":"Biochem. Soc. Trans."},{"key":"ref_64","doi-asserted-by":"crossref","first-page":"325","DOI":"10.1086\/422762","article-title":"Intergenerational instability of the expanded CTG repeat in the DMPK gene: Studies in human gametes and preimplantation embryos","volume":"75","author":"Sermon","year":"2004","journal-title":"Am. J. Hum. Genet."},{"key":"ref_65","doi-asserted-by":"crossref","first-page":"196","DOI":"10.3389\/fnagi.2014.00196","article-title":"RNA transcription and maturation in skeletal muscle cells are similarly impaired in myotonic dystrophy and sarcopenia: The ultrastructural evidence","volume":"6","author":"Malatesta","year":"2014","journal-title":"Front. Aging Neurosci."},{"key":"ref_66","doi-asserted-by":"crossref","first-page":"862","DOI":"10.1002\/ana.23992","article-title":"Splicing biomarkers of disease severity in myotonic dystrophy","volume":"74","author":"Nakamori","year":"2013","journal-title":"Ann. Neurol."},{"key":"ref_67","doi-asserted-by":"crossref","first-page":"4439","DOI":"10.1093\/emboj\/19.17.4439","article-title":"Recruitment of human muscleblind proteins to (CUG)(n) expansions associated with myotonic dystrophy","volume":"19","author":"Miller","year":"2000","journal-title":"EMBO J."},{"key":"ref_68","doi-asserted-by":"crossref","first-page":"594","DOI":"10.1016\/j.bbadis.2014.05.019","article-title":"Myotonic dystrophies: An update on clinical aspects, genetic, pathology, and molecular pathomechanisms","volume":"1852","author":"Meola","year":"2015","journal-title":"Biochim. Biophys. Acta"},{"key":"ref_69","doi-asserted-by":"crossref","first-page":"4271","DOI":"10.1038\/sj.emboj.7601296","article-title":"Interaction of musleblind, CUG-BP1 and hnRNP H proteins in DM1-associated aberrant IR splicing","volume":"25","author":"Paul","year":"2006","journal-title":"EMBO J."},{"key":"ref_70","doi-asserted-by":"crossref","first-page":"e18","DOI":"10.4081\/ejh.2009.e18","article-title":"RNA\/MBNL1-containing foci in myoblast nuclei from patients affected by myotonic dystrophy type 2: An immunocytochemical study","volume":"53","author":"Perdoni","year":"2009","journal-title":"Eur. J. Histochem."},{"key":"ref_71","doi-asserted-by":"crossref","first-page":"13915","DOI":"10.1073\/pnas.0905780106","article-title":"Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy","volume":"106","author":"Mulders","year":"2009","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_72","doi-asserted-by":"crossref","unstructured":"Sardone, V., Zhou, H., Muntoni, F., Ferlini, A., and Falzarano, M.S. (2017). Antisense oligonucleotide-based therapy for neuromuscular disease. Molecules, 22.","DOI":"10.3390\/molecules22040563"},{"key":"ref_73","doi-asserted-by":"crossref","first-page":"11748","DOI":"10.1073\/pnas.0604970103","article-title":"Reversal of RNA missplicing and myotonia after muscleblind overexpression in a mouse poly(CUG) model for myotonic dystrophy","volume":"103","author":"Kanadia","year":"2006","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_74","doi-asserted-by":"crossref","first-page":"16068","DOI":"10.1073\/pnas.0901824106","article-title":"A simple ligand that selectively targets CUG trinucleotide repeats and inhibits MBNL protein binding","volume":"106","author":"Arambula","year":"2009","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_75","doi-asserted-by":"crossref","first-page":"856","DOI":"10.1021\/cb200408a","article-title":"Rationally designed small molecules targeting the RNA that causes myotonic dystrophy type 1 are potently bioactive","volume":"7","author":"Hoskins","year":"2012","journal-title":"ACS Chem. Biol."},{"key":"ref_76","doi-asserted-by":"crossref","first-page":"891","DOI":"10.1016\/S1474-4422(12)70204-1","article-title":"The myotonic dystrophies: Molecular, clinical, and therapeutic challenges","volume":"11","author":"Udd","year":"2012","journal-title":"Lancet Neurol."},{"key":"ref_77","doi-asserted-by":"crossref","first-page":"637","DOI":"10.1002\/ana.23982","article-title":"Eteplirsen for the treatment of Duchenne muscular dystrophy","volume":"74","author":"Mendell","year":"2013","journal-title":"Ann. Neurol."},{"key":"ref_78","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1016\/j.nmd.2017.10.004","article-title":"A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy","volume":"28","author":"Goemans","year":"2018","journal-title":"Neuromuscul. Disord."},{"key":"ref_79","doi-asserted-by":"crossref","first-page":"1513","DOI":"10.1056\/NEJMoa1011367","article-title":"Systemic administration of PRO051 in Duchenne\u2019s muscular dystrophy","volume":"365","author":"Goemans","year":"2011","journal-title":"N. Engl. J. Med."},{"key":"ref_80","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1124\/jpet.115.226969","article-title":"Identification and characterization of modified antisense oligonucleotides targeting DMPK in mice and nonhuman primates for the treatment of myotonic dystrophy type 1s","volume":"355","author":"Pandey","year":"2015","journal-title":"J. Pharmacol. Exp. Ther."},{"key":"ref_81","doi-asserted-by":"crossref","first-page":"4739","DOI":"10.1172\/JCI128205","article-title":"Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice","volume":"129","author":"Klein","year":"2019","journal-title":"J. Clin. Investig."},{"key":"ref_82","doi-asserted-by":"crossref","first-page":"eaav4324","DOI":"10.1126\/sciadv.aav4324","article-title":"CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells","volume":"5","author":"Min","year":"2019","journal-title":"Sci. Adv."},{"key":"ref_83","doi-asserted-by":"crossref","first-page":"86","DOI":"10.1126\/science.aau1549","article-title":"Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy","volume":"362","author":"Amoasii","year":"2018","journal-title":"Science"},{"key":"ref_84","doi-asserted-by":"crossref","first-page":"343","DOI":"10.3233\/JND-150116","article-title":"Genome editing gene therapy for Duchenne muscular dystrophy","volume":"2","author":"Hotta","year":"2015","journal-title":"J. Neuromuscul. Dis."},{"key":"ref_85","doi-asserted-by":"crossref","first-page":"eaan8081","DOI":"10.1126\/scitranslmed.aan8081","article-title":"Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy","volume":"29","author":"Amoasii","year":"2017","journal-title":"Sci. Transl. Med."},{"key":"ref_86","doi-asserted-by":"crossref","first-page":"8275","DOI":"10.1093\/nar\/gky548","article-title":"Efficient CRISPR\/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells","volume":"46","author":"Dastidar","year":"2018","journal-title":"Nucleic Acids Res."},{"key":"ref_87","doi-asserted-by":"crossref","first-page":"706","DOI":"10.1002\/ana.1023","article-title":"Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations","volume":"49","author":"Wagner","year":"2001","journal-title":"Ann. Neurol."},{"key":"ref_88","doi-asserted-by":"crossref","first-page":"375","DOI":"10.1172\/JCI7866","article-title":"Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice","volume":"104","author":"Cordier","year":"1999","journal-title":"J. Clin. Investig."},{"key":"ref_89","doi-asserted-by":"crossref","unstructured":"Vitiello, L., Tibaudo, L., Pegoraro, E., Bello, L., and Canton, M. (2019). Teaching an old molecule new tricks: Drug repositioning for Duchenne muscular dystrophy. Int. J. Mol. Sci., 20.","DOI":"10.3390\/ijms20236053"},{"key":"ref_90","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1038\/nature05756","article-title":"PTC124 targets genetic disorders caused by nonsense mutations","volume":"447","author":"Welch","year":"2007","journal-title":"Nature"},{"key":"ref_91","doi-asserted-by":"crossref","first-page":"1489","DOI":"10.1016\/S0140-6736(17)31611-2","article-title":"Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): A multicentre, randomised, double-blind, placebo-controlled, phase 3 trial","volume":"390","author":"McDonald","year":"2017","journal-title":"Lancet"},{"key":"ref_92","doi-asserted-by":"crossref","first-page":"5770","DOI":"10.1021\/acs.jmedchem.5b00356","article-title":"Biological efficacy and toxicity of diamidines in myotonic dystrophy type 1 models","volume":"58","author":"Siboni","year":"2015","journal-title":"J. Med. Chem."},{"key":"ref_93","doi-asserted-by":"crossref","first-page":"2708","DOI":"10.1021\/acschembio.8b00646","article-title":"Furamidine rescues myotonic dystrophy type I associated mis-splicing through multiple mechanisms","volume":"13","author":"Jenquin","year":"2018","journal-title":"ACS Chem. Biol."},{"key":"ref_94","doi-asserted-by":"crossref","unstructured":"Reddy, K., Jenquin, J.R., Cleary, J.D., and Berglund, J.A. (2019). Mitigating RNA toxicity in myotonic dystrophy using small molecules. Int. J. Mol. Sci., 20.","DOI":"10.3390\/ijms20164017"},{"key":"ref_95","doi-asserted-by":"crossref","first-page":"247","DOI":"10.1021\/acsptsci.9b00020","article-title":"Combination treatment of erythromycin and furamidine provides additive and synergistic rescue of mis-splicing in myotonic dystrophy type 1 models","volume":"2","author":"Jenquin","year":"2019","journal-title":"ACS Pharmacol. Transl. Sci."},{"key":"ref_96","doi-asserted-by":"crossref","first-page":"3056","DOI":"10.1093\/hmg\/ddx190","article-title":"A flow cytometry-based screen identifies MBNL1 modulators that rescue splicing defects in myotonic dystrophy type I","volume":"26","author":"Zhang","year":"2017","journal-title":"Hum. Mol. Genet."},{"key":"ref_97","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1038\/nature15818","article-title":"Gene therapy returns to centre stage","volume":"526","author":"Naldini","year":"2015","journal-title":"Nature"},{"key":"ref_98","doi-asserted-by":"crossref","first-page":"457","DOI":"10.1038\/ni0610-457","article-title":"20 years of gene therapy for SCID","volume":"11","author":"Fischer","year":"2010","journal-title":"Nat. Immunol."},{"key":"ref_99","doi-asserted-by":"crossref","first-page":"eaan4672","DOI":"10.1126\/science.aan4672","article-title":"Gene therapy comes of age","volume":"359","author":"Dunbar","year":"2018","journal-title":"Science"},{"key":"ref_100","doi-asserted-by":"crossref","first-page":"2290","DOI":"10.1172\/JCI25424","article-title":"Antisense oligonucleotide therapy for neurodegenerative disease","volume":"116","author":"Smith","year":"2006","journal-title":"J. Clin. Investig."},{"key":"ref_101","doi-asserted-by":"crossref","first-page":"2075","DOI":"10.1016\/j.ymthe.2017.06.002","article-title":"RNase H1-dependent antisense oligonucleotides are robustly active in directing RNA cleavage in both the cytoplasm and the nucleus","volume":"25","author":"Liang","year":"2017","journal-title":"Mol. Ther."},{"key":"ref_102","doi-asserted-by":"crossref","first-page":"46","DOI":"10.1016\/j.addr.2015.01.008","article-title":"Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides","volume":"87","author":"Geary","year":"2015","journal-title":"Adv. Drug Deliv. Rev."},{"key":"ref_103","doi-asserted-by":"crossref","first-page":"3892","DOI":"10.1093\/nar\/gkw144","article-title":"Hsp90 protein interacts with phosphorothioate oligonucleotides containing hydrophobic 2\u2032-modifications and enhances antisense activity","volume":"44","author":"Liang","year":"2016","journal-title":"Nucleic Acids Res."},{"key":"ref_104","doi-asserted-by":"crossref","unstructured":"He, X.-Y., Wang, J., Lu, D.-D., and Wang, S.-Q. (2018). Synthesis and antisense properties of 2\u2032\u03b2-F-arabinouridine modified oligonucleotides with 4\u2032-C-OMe substituent. Molecules, 23.","DOI":"10.3390\/molecules23092374"},{"key":"ref_105","doi-asserted-by":"crossref","first-page":"5366","DOI":"10.1093\/nar\/gky397","article-title":"Identifying and avoiding off-target effects of RNase H-dependent antisense oligonucleotides in mice","volume":"46","author":"Hagedorn","year":"2018","journal-title":"Nucleic Acids Res."},{"key":"ref_106","doi-asserted-by":"crossref","first-page":"305","DOI":"10.1089\/nat.2019.0805","article-title":"The pharmacokinetics of 2\u2032-O-methyl phosphorothioate antisense oligonucleotides: Experiences from developing exon skipping therapies for Duchenne muscular dystrophy","volume":"29","author":"Bosgra","year":"2019","journal-title":"Nucleic Acid Ther."},{"key":"ref_107","doi-asserted-by":"crossref","first-page":"553","DOI":"10.1007\/978-1-4939-8651-4_36","article-title":"Exon skipping using antisense oligonucleotides for laminin-alpha2-deficient muscular dystrophy","volume":"1828","author":"Hara","year":"2018","journal-title":"Methods Mol. Biol."},{"key":"ref_108","doi-asserted-by":"crossref","first-page":"1578","DOI":"10.1126\/science.2662404","article-title":"The molecular basis of muscular dystrophy in the mdx mouse: A point mutation","volume":"244","author":"Sicinski","year":"1989","journal-title":"Science"},{"key":"ref_109","doi-asserted-by":"crossref","first-page":"918","DOI":"10.1016\/S1474-4422(09)70211-X","article-title":"Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: A single-blind, placebo-controlled, dose-escalation, proof-of-concept study","volume":"8","author":"Kinali","year":"2009","journal-title":"Lancet Neurol."},{"key":"ref_110","doi-asserted-by":"crossref","first-page":"442","DOI":"10.1016\/j.omtn.2018.09.017","article-title":"NS-065\/NCNP-01: An antisense oligonucleotide for potential treatment of exon 53 skipping in Duchenne muscular dystrophy","volume":"13","author":"Watanabe","year":"2018","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"ref_111","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1089\/hum.2012.212","article-title":"Antisense oligonucleotides: Rising stars in eliminating RNA toxicity in myotonic dystrophy","volume":"24","author":"Gao","year":"2013","journal-title":"Hum. Gene Ther."},{"key":"ref_112","doi-asserted-by":"crossref","unstructured":"Huguet, A., Medja, F., Nicole, A., Vignaud, A., Guiraud-Dogan, C., Ferry, A., Decostre, V., Hogrel, J.Y., Metzger, F., and Hoeflich, A. (2012). Molecular, physiological, and motor performance defects in DMSXL mice carrying >1,000 CTG repeats from the human DM1 locus. PLoS Genet., 8.","DOI":"10.1371\/journal.pgen.1003043"},{"key":"ref_113","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1038\/nature11362","article-title":"Targeting nuclear RNA for in vivo correction of myotonic dystrophy","volume":"488","author":"Wheeler","year":"2012","journal-title":"Nature"},{"key":"ref_114","doi-asserted-by":"crossref","first-page":"465","DOI":"10.1016\/j.omtn.2017.05.007","article-title":"Targeting DMPK with antisense oligonucleotide improves muscle strength in myotonic dystrophy type 1 mice","volume":"7","author":"Jauvin","year":"2017","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"ref_115","doi-asserted-by":"crossref","first-page":"6244","DOI":"10.1038\/ncomms7244","article-title":"Multiplex CRISPR\/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy","volume":"6","author":"Ousterout","year":"2015","journal-title":"Nat. Commun."},{"key":"ref_116","doi-asserted-by":"crossref","first-page":"523","DOI":"10.1038\/mt.2014.234","article-title":"Correction of dystrophin expression in cells from Duchenne Muscular Dystrophy patients through genomic excision of exon 51 by Zinc Finger Nucleases","volume":"23","author":"Ousterout","year":"2015","journal-title":"Mol. Ther."},{"key":"ref_117","doi-asserted-by":"crossref","first-page":"1718","DOI":"10.1038\/mt.2013.111","article-title":"Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne Muscular Dystrophy patients","volume":"21","author":"Ousterout","year":"2013","journal-title":"Mol. Ther."},{"key":"ref_118","doi-asserted-by":"crossref","first-page":"1258096","DOI":"10.1126\/science.1258096","article-title":"Genome editing. The new frontier of genome engineering with CRISPR-Cas9","volume":"346","author":"Doudna","year":"2014","journal-title":"Science"},{"key":"ref_119","doi-asserted-by":"crossref","first-page":"e1472","DOI":"10.1002\/wnan.1472","article-title":"Nanotherapy for Duchenne muscular dystrophy","volume":"10","author":"Nance","year":"2018","journal-title":"Wiley Interdiscip. Rev. Nanomed. Nanobiotechnol."},{"key":"ref_120","doi-asserted-by":"crossref","first-page":"67","DOI":"10.1038\/nrmicro.2015.14","article-title":"CRISPR-Cas adaptation: Insights into the mechanism of action","volume":"14","author":"Amitai","year":"2016","journal-title":"Nat. Rev. Microbiol."},{"key":"ref_121","doi-asserted-by":"crossref","first-page":"481","DOI":"10.1038\/nature14592","article-title":"Engineered CRISPR-Cas9 nucleases with altered PAM specificities","volume":"523","author":"Kleinstiver","year":"2015","journal-title":"Nature"},{"key":"ref_122","doi-asserted-by":"crossref","first-page":"2617","DOI":"10.1016\/j.ymthe.2018.09.003","article-title":"Therapeutic genome editing for myotonic dystrophy type 1 using CRISPR\/Cas9","volume":"26","author":"Wang","year":"2018","journal-title":"Mol. Ther."},{"key":"ref_123","doi-asserted-by":"crossref","first-page":"317","DOI":"10.1007\/s40259-017-0234-5","article-title":"Adeno-associated virus (AAV) as a vector for gene therapy","volume":"31","author":"Naso","year":"2017","journal-title":"BioDrugs"},{"key":"ref_124","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1038\/nature17946","article-title":"Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage","volume":"533","author":"Komor","year":"2016","journal-title":"Nature"},{"key":"ref_125","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1038\/nature24644","article-title":"Programmable base editing of A\u2022T to G\u2022C in genomic DNA without DNA cleavage","volume":"551","author":"Gaudelli","year":"2017","journal-title":"Nature"},{"key":"ref_126","doi-asserted-by":"crossref","first-page":"382","DOI":"10.1126\/science.aax7063","article-title":"A Cytosine deaminase for programmable single-base RNA editing","volume":"365","author":"Abudayyeh","year":"2019","journal-title":"Science"},{"key":"ref_127","doi-asserted-by":"crossref","first-page":"536","DOI":"10.1038\/nbt.4148","article-title":"Adenine base editing in mouse embryos and an adult mouse model of Duchenne Muscular Dystrophy","volume":"36","author":"Ryu","year":"2018","journal-title":"Nat Biotechnol"},{"key":"ref_128","doi-asserted-by":"crossref","first-page":"1979","DOI":"10.1038\/s41467-020-15892-8","article-title":"Chemical modifications of adenine base editor mRNA and guide RNA expand its application scope","volume":"11","author":"Jiang","year":"2020","journal-title":"Nat Commun"},{"key":"ref_129","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1038\/s41586-019-1711-4","article-title":"Search-and-replace genome editing without double-strand breaks or donor DNA","volume":"576","author":"Anzalone","year":"2019","journal-title":"Nature"},{"key":"ref_130","doi-asserted-by":"crossref","first-page":"41","DOI":"10.1038\/nrd.2018.168","article-title":"Drug repurposing: Progress, challenges and recommendations","volume":"18","author":"Pushpakom","year":"2019","journal-title":"Nat. Rev. Drug Discov."},{"key":"ref_131","doi-asserted-by":"crossref","first-page":"936","DOI":"10.1038\/s41416-018-0045-6","article-title":"New opportunities for kinase drug repurposing and target discovery","volume":"118","author":"Knapp","year":"2018","journal-title":"Br. J. Cancer"},{"key":"ref_132","doi-asserted-by":"crossref","first-page":"1695","DOI":"10.1007\/s00109-019-01847-0","article-title":"Serum starvation enhances nonsense mutation readthrough","volume":"97","author":"Wittenstein","year":"2019","journal-title":"J. Mol. Med."},{"key":"ref_133","doi-asserted-by":"crossref","first-page":"373","DOI":"10.1093\/hmg\/ddx409","article-title":"Aminoglycoside-mediated promotion of translation readthrough occurs through a non-stochastic mechanism that competes with translation termination","volume":"27","author":"Chowdhury","year":"2018","journal-title":"Hum. Mol. Genet."},{"key":"ref_134","doi-asserted-by":"crossref","unstructured":"Shimizu-Motohashi, Y., Komaki, H., Motohashi, N., Takeda, S., Yokota, T., and Aoki, Y. (2019). Restoring dystrophin expression in Duchenne muscular dystrophy: Current status of therapeutic approaches. J. Pers. Med., 9.","DOI":"10.3390\/jpm9010001"},{"key":"ref_135","doi-asserted-by":"crossref","first-page":"223","DOI":"10.1111\/bcp.13439","article-title":"Adverse effects of a single dose of gentamicin in adults: A systematic review","volume":"84","author":"Hayward","year":"2018","journal-title":"Br. J. Clin. Pharmacol."},{"key":"ref_136","doi-asserted-by":"crossref","unstructured":"Friesen, W.J., Johnson, B., Sierra, J., Zhuo, J., Vazirani, P., Xue, X., Tomizawa, Y., Baiazitov, R., Morrill, C., and Ren, H. (2018). The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential. PLoS ONE, 13.","DOI":"10.1371\/journal.pone.0206158"},{"key":"ref_137","doi-asserted-by":"crossref","unstructured":"Gonzalez, \u00c0.L., Konieczny, P., Llamusi, B., Delgado-Pinar, E., Borrell, J.I., Teixid\u00f3, J., Garc\u00eda-Espa\u00f1a, E., P\u00e9rez-Alonso, M., Estrada-Tejedor, R., and Artero, R. (2017). In silico discovery of substituted pyrido[2,3-d] pyrimidines and pentamidine-like compounds with biological activity in myotonic dystrophy models. PLoS ONE, 12.","DOI":"10.1371\/journal.pone.0178931"},{"key":"ref_138","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.actatropica.2014.02.012","article-title":"Pentamidine exerts in vitro and in vivo anti Trypanosoma cruzi activity and inhibits the polyamine transport in Trypanosoma cruzi","volume":"134","author":"Miranda","year":"2014","journal-title":"Acta Trop."},{"key":"ref_139","doi-asserted-by":"crossref","first-page":"2528","DOI":"10.1021\/cb400431f","article-title":"Reducing levels of toxic RNA with small molecules","volume":"8","author":"Coonrod","year":"2013","journal-title":"ACS Chem. Biol."},{"key":"ref_140","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1016\/j.addr.2015.11.001","article-title":"Extracellular matrix structure","volume":"97","author":"Theocharis","year":"2016","journal-title":"Adv. Drug Deliv. Rev."},{"key":"ref_141","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1089\/10430340150504046","article-title":"Efficient expression of naked DNA delivered intraarterially to limb muscles of nonhuman primates","volume":"12","author":"Zhang","year":"2001","journal-title":"Hum. Gene Ther."},{"key":"ref_142","doi-asserted-by":"crossref","first-page":"386","DOI":"10.1016\/j.ymthe.2004.05.004","article-title":"A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs","volume":"10","author":"Hagstrom","year":"2004","journal-title":"Mol. Ther."},{"key":"ref_143","doi-asserted-by":"crossref","first-page":"1600","DOI":"10.1126\/science.8128245","article-title":"Biodegradable long-circulating polymeric nanospheres","volume":"263","author":"Gref","year":"1994","journal-title":"Science"},{"key":"ref_144","doi-asserted-by":"crossref","first-page":"703","DOI":"10.2217\/17435889.3.5.703","article-title":"Clearance properties of nano-sized particles and molecules as imaging agents: Considerations and caveats","volume":"3","author":"Longmire","year":"2008","journal-title":"Nanomedicine"},{"key":"ref_145","doi-asserted-by":"crossref","first-page":"487","DOI":"10.1016\/j.nantod.2015.06.006","article-title":"Nanoparticle uptake: The phagocyte problem","volume":"10","author":"Gustafson","year":"2015","journal-title":"Nano Today"},{"key":"ref_146","doi-asserted-by":"crossref","first-page":"941","DOI":"10.1038\/nbt.3330","article-title":"Principles of nanoparticle design for overcoming biological barriers to drug delivery","volume":"33","author":"Blanco","year":"2015","journal-title":"Nat. Biotechnol."},{"key":"ref_147","doi-asserted-by":"crossref","first-page":"3137","DOI":"10.2147\/IJN.S129300","article-title":"Protein corona: A new approach for nanomedicine design","volume":"12","author":"Nguyen","year":"2017","journal-title":"Int. J. Nanomed."},{"key":"ref_148","doi-asserted-by":"crossref","first-page":"772","DOI":"10.1038\/nnano.2013.181","article-title":"Rapid formation of plasma protein corona critically affects nanoparticle pathophysiology","volume":"8","author":"Tenzer","year":"2013","journal-title":"Nat. Nanotechnol."},{"key":"ref_149","doi-asserted-by":"crossref","first-page":"7541","DOI":"10.1016\/j.vaccine.2012.10.050","article-title":"Nanoparticle size influences the magnitude and quality of mucosal immune responses after intranasal immunization","volume":"30","author":"Stano","year":"2012","journal-title":"Vaccine"},{"key":"ref_150","doi-asserted-by":"crossref","first-page":"673","DOI":"10.2217\/nnm.16.5","article-title":"The effect of nanoparticle size on in vivo pharmacokinetics and cellular interaction","volume":"11","author":"Hoshyar","year":"2016","journal-title":"Nanomedicine"},{"key":"ref_151","doi-asserted-by":"crossref","first-page":"58","DOI":"10.1016\/j.taap.2016.01.002","article-title":"Enzymatic oxidative biodegradation of nanoparticles: Mechanisms, significance and applications","volume":"299","author":"Vlasova","year":"2016","journal-title":"Toxicol. Appl. Pharmacol."},{"key":"ref_152","doi-asserted-by":"crossref","first-page":"1313","DOI":"10.1002\/cphy.c170052","article-title":"Immunobiology of inherited muscular dystrophies","volume":"8","author":"Tidball","year":"2018","journal-title":"Compr. Physiol."},{"key":"ref_153","doi-asserted-by":"crossref","first-page":"100069","DOI":"10.1016\/j.mtbio.2020.100069","article-title":"Advances in biomaterials for skeletal muscle engineering and obstacles still to overcome","volume":"7","author":"Smoak","year":"2020","journal-title":"Mater. Today Bio"},{"key":"ref_154","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1016\/j.addr.2015.03.008","article-title":"Antisense oligonucleotides in therapy for neurodegenerative disorders","volume":"87","author":"Evers","year":"2015","journal-title":"Adv. Drug Deliv. Rev."},{"key":"ref_155","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1016\/j.jconrel.2015.12.032","article-title":"Materials for non-viral intracellular delivery of messenger RNA therapeutics","volume":"240","author":"Kauffman","year":"2016","journal-title":"J. Control. Release"},{"key":"ref_156","doi-asserted-by":"crossref","first-page":"527","DOI":"10.1021\/acscombsci.6b00073","article-title":"Cellular delivery of RNA nanoparticles","volume":"18","author":"Parlea","year":"2016","journal-title":"ACS Comb. Sci."},{"key":"ref_157","doi-asserted-by":"crossref","unstructured":"Wahane, A., Waghmode, A., Kapphahn, A., Dhuri, K., Gupta, A., and Bahal, R. (2020). Role of lipid-based and polymer-based non-viral vectors in nucleic acid delivery for next-generation gene therapy. Molecules, 25.","DOI":"10.3390\/molecules25122866"},{"key":"ref_158","doi-asserted-by":"crossref","first-page":"364","DOI":"10.1016\/j.jconrel.2017.11.021","article-title":"Nanoparticle formulation improves doxorubicin efficacy by enhancing host antitumor immunity","volume":"269","author":"Mastria","year":"2018","journal-title":"J. Control. Release"},{"key":"ref_159","doi-asserted-by":"crossref","first-page":"862","DOI":"10.1124\/pr.120.019554","article-title":"RNA drugs and RNA targets for small molecules: Principles, progress, and challenges","volume":"72","author":"Yu","year":"2020","journal-title":"Pharmacol. Rev."},{"key":"ref_160","doi-asserted-by":"crossref","first-page":"108","DOI":"10.1208\/s12248-018-0267-9","article-title":"Nanoparticle based delivery of CRISPR\/Cas9 genome editing therapeutics","volume":"20","author":"Givens","year":"2018","journal-title":"AAPS J."},{"key":"ref_161","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1016\/j.jconrel.2017.09.012","article-title":"Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications","volume":"266","author":"Liu","year":"2017","journal-title":"J. Control. Release"},{"key":"ref_162","doi-asserted-by":"crossref","first-page":"239","DOI":"10.1146\/annurev-med-081117-010451","article-title":"CRISPR correction of Duchenne muscular dystrophy","volume":"27","author":"Min","year":"2019","journal-title":"Annu. Rev. Med."},{"key":"ref_163","doi-asserted-by":"crossref","unstructured":"Williams, J.H., Schray, R.C., Sirsi, S.R., and Lutz, G.J. (2008). Nanopolymers improve delivery of exon skipping oligonucleotides and concomitant dystrophin expression in skeletal muscle of mdx mice. BMC Biotechnol., 8.","DOI":"10.1186\/1472-6750-8-35"},{"key":"ref_164","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1186\/1477-3155-7-1","article-title":"Formulation of polylactide-co-glycolic acid nanospheres for encapsulation and sustained release of poly(ethylene imine)-poly(ethylene glycol) copolymers complexed to oligonucleotides","volume":"7","author":"Sirsi","year":"2009","journal-title":"J. Nanobiotechnol."},{"key":"ref_165","doi-asserted-by":"crossref","first-page":"210","DOI":"10.1038\/mt.2012.236","article-title":"Polyethylenimine-modified Pluronics (PCMs) improve morpholino oligomer delivery in cell culture and dystrophic mdx mice","volume":"21","author":"Wang","year":"2013","journal-title":"Mol. Ther."},{"key":"ref_166","doi-asserted-by":"crossref","first-page":"52","DOI":"10.1038\/gt.2013.57","article-title":"Pluronic\u2013PEI copolymers enhance exon-skipping of 2\u2032-O-methyl phosphorothioate oligonucleotide in cell culture and dystrophic mdx mice","volume":"21","author":"Wang","year":"2014","journal-title":"Gene Ther."},{"key":"ref_167","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1016\/j.jconrel.2008.10.020","article-title":"Polymersome delivery of siRNA and antisense oligonucleotides","volume":"134","author":"Kim","year":"2009","journal-title":"J. Control. Release"},{"key":"ref_168","doi-asserted-by":"crossref","first-page":"820","DOI":"10.1038\/mt.2009.8","article-title":"Cationic PMMA nanoparticles bind and deliver antisense oligoribonucleotides allowing restoration of dystrophin expression in the mdx mouse","volume":"17","author":"Rimessi","year":"2009","journal-title":"Mol. Ther."},{"key":"ref_169","doi-asserted-by":"crossref","first-page":"432","DOI":"10.1038\/gt.2009.145","article-title":"Dystrophin restoration in skeletal, heart and skin arrector pili smooth muscle of mdx mice by ZM2 NP\u2013AON complexes","volume":"17","author":"Ferlini","year":"2010","journal-title":"Gene Ther."},{"key":"ref_170","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1155\/2012\/897076","article-title":"Persistent dystrophin protein restoration 90 days after a course of intraperitoneally administered naked 2\u2032OMePS AON and ZM2 NP-AON complexes in mdx Mice","volume":"2012","author":"Bassi","year":"2012","journal-title":"J. Biomed. Biotechnol."},{"key":"ref_171","doi-asserted-by":"crossref","first-page":"e341","DOI":"10.1038\/mtna.2016.51","article-title":"Poly(ester amine) composed of polyethylenimine and Pluronic enhance delivery of antisense oligonucleotides in vitro and in dystrophic mdx mice","volume":"5","author":"Wang","year":"2016","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"ref_172","doi-asserted-by":"crossref","first-page":"837","DOI":"10.1021\/bc200674e","article-title":"Tris[2-(acryloyloxy)ethyl]isocyanurate cross-linked low-molecular-weight polyethylenimine as gene delivery carriers in cell culture and dystrophic mdx mice","volume":"23","author":"Wang","year":"2012","journal-title":"Bioconjug. Chem."},{"key":"ref_173","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1016\/j.jconrel.2009.12.014","article-title":"Polyplex nanomicelle promotes hydrodynamic gene introduction to skeletal muscle","volume":"143","author":"Itaka","year":"2010","journal-title":"J. Control. Release"},{"key":"ref_174","doi-asserted-by":"crossref","first-page":"1254","DOI":"10.1038\/sj.gt.3301794","article-title":"Enhanced gene expression in mouse muscle by sustained release of plasmid DNA using PPE-EA as a carrier","volume":"9","author":"Wang","year":"2002","journal-title":"Gene Ther."},{"key":"ref_175","doi-asserted-by":"crossref","first-page":"1126","DOI":"10.1038\/gt.2008.24","article-title":"Atelocollagen-mediated local and systemic applications of myostatin-targeting siRNA increase skeletal muscle mass","volume":"15","author":"Kinouchi","year":"2008","journal-title":"Gene Ther."},{"key":"ref_176","doi-asserted-by":"crossref","first-page":"1985","DOI":"10.2147\/IJN.S125521","article-title":"The complex of PAMAM-OH dendrimer with angiotensin (1-7) prevented the disuse-induced skeletal muscle atrophy in mice","volume":"12","author":"Abrigo","year":"2017","journal-title":"Int. J. Nanomed."},{"key":"ref_177","doi-asserted-by":"crossref","first-page":"1053","DOI":"10.1021\/mp4004755","article-title":"Bubble liposomes and ultrasound exposure improve localized morpholino oligomer delivery into the skeletal muscles of dystrophic mdx mice","volume":"11","author":"Negishi","year":"2014","journal-title":"Mol. Pharm."},{"key":"ref_178","doi-asserted-by":"crossref","first-page":"2242","DOI":"10.1038\/srep02242","article-title":"Ultrasound-enhanced delivery of morpholino with bubble liposomes ameliorates the myotonia of myotonic dystrophy model mice","volume":"3","author":"Koebis","year":"2013","journal-title":"Sci. Rep."},{"key":"ref_179","first-page":"2943","article-title":"Nanolipodendrosome-loaded glatiramer acetate and myogenic differentiation 1 as augmentation therapeutic strategy approaches in muscular dystrophy","volume":"8","author":"Afzal","year":"2013","journal-title":"Int. J. Nanomed."},{"key":"ref_180","doi-asserted-by":"crossref","first-page":"34","DOI":"10.1016\/j.nano.2018.11.012","article-title":"Liposomal steroid nano-drug is superior to steroids as-is in mdx mouse model of Duchenne muscular mystrophy","volume":"16","author":"Turjeman","year":"2019","journal-title":"Nanomedicine"},{"key":"ref_181","doi-asserted-by":"crossref","first-page":"712","DOI":"10.1248\/bpb.34.712","article-title":"Effective drug delivery system for Duchenne muscular dystrophy using hybrid liposomes including gentamicin along with reduced toxicity","volume":"34","author":"Yukihara","year":"2011","journal-title":"Biol. Pharm. Bull."},{"key":"ref_182","doi-asserted-by":"crossref","first-page":"2047","DOI":"10.1096\/fj.13-237388","article-title":"Rapamycin nanoparticles target defective autophagy in muscular dystrophy to enhance both strength and cardiac function","volume":"28","author":"Bibee","year":"2014","journal-title":"FASEB J."},{"key":"ref_183","doi-asserted-by":"crossref","first-page":"3232","DOI":"10.1038\/s41467-020-17029-3","article-title":"Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing","volume":"11","author":"Wei","year":"2020","journal-title":"Nat. Commun."},{"key":"ref_184","doi-asserted-by":"crossref","first-page":"889","DOI":"10.1038\/s41551-017-0137-2","article-title":"Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair","volume":"1","author":"Lee","year":"2017","journal-title":"Nat. Biomed. Eng."},{"key":"ref_185","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1089\/nat.2013.0454","article-title":"A chemical view of oligonucleotides for exon skipping and related drug applications","volume":"24","author":"Gait","year":"2014","journal-title":"Nucleic Acid Ther."},{"key":"ref_186","doi-asserted-by":"crossref","first-page":"26","DOI":"10.1007\/s41061-017-0112-0","article-title":"History of polymeric gene delivery systems","volume":"375","author":"Zhang","year":"2017","journal-title":"Top. Curr. Chem."},{"key":"ref_187","doi-asserted-by":"crossref","first-page":"7297","DOI":"10.1073\/pnas.92.16.7297","article-title":"A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: Polyethylenimine","volume":"92","author":"Boussif","year":"1995","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_188","doi-asserted-by":"crossref","first-page":"1009","DOI":"10.1038\/nm897","article-title":"Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse","volume":"9","author":"Lu","year":"2003","journal-title":"Nat. Med."},{"key":"ref_189","doi-asserted-by":"crossref","first-page":"131","DOI":"10.1038\/sj.gt.3301874","article-title":"Non-viral gene delivery in skeletal muscle: A protein factory","volume":"10","author":"Lu","year":"2003","journal-title":"Gene Ther."},{"key":"ref_190","doi-asserted-by":"crossref","first-page":"88","DOI":"10.1016\/j.ymthe.2005.11.025","article-title":"Induction of dystrophin expression by exon skipping in mdx mice following intramuscular injection of antisense oligonucleotides complexed with PEG\u2013PEI copolymers","volume":"14","author":"Williams","year":"2006","journal-title":"Mol. Ther."},{"key":"ref_191","doi-asserted-by":"crossref","first-page":"141","DOI":"10.1007\/978-1-59745-237-3_9","article-title":"PEG-PEI copolymers for oligonucleotide delivery to Cells and tissues","volume":"433","author":"Lutz","year":"2008","journal-title":"Methods Mol. Biol."},{"key":"ref_192","doi-asserted-by":"crossref","first-page":"5655","DOI":"10.1016\/j.vaccine.2006.05.058","article-title":"DNA prime and protein boost immunization with innovative polymeric cationic core-shell nanoparticles elicits broad immune responses and strongly enhance cellular responses of HIV-1 tat DNA vaccination","volume":"24","author":"Castaldello","year":"2006","journal-title":"Vaccine"},{"key":"ref_193","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1089\/oli.1.1997.7.187","article-title":"Morpholino antisense oligomers: Design, preparation, and properties","volume":"7","author":"Summerton","year":"1997","journal-title":"Antisense Nucleic Acid Drug Dev."},{"key":"ref_194","first-page":"e1393","article-title":"Antisense oligonucleotides: Treatment strategies and cellular internalization","volume":"3","author":"Miller","year":"2016","journal-title":"RNA Dis."},{"key":"ref_195","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1089\/nat.2014.0511","article-title":"Peptide nanoparticle delivery of charge-neutral splice-switching morpholino oligonucleotides","volume":"25","author":"Zaghloul","year":"2015","journal-title":"Nucleic Acid Ther."},{"key":"ref_196","doi-asserted-by":"crossref","first-page":"130","DOI":"10.1016\/j.jconrel.2006.09.008","article-title":"Gene delivery by combination of novel liposomal bubbles with perfluoropropane and ultrasound","volume":"117","author":"Suzuki","year":"2007","journal-title":"J. Control. Release"},{"key":"ref_197","doi-asserted-by":"crossref","first-page":"124","DOI":"10.1016\/j.jconrel.2008.08.019","article-title":"Delivery of siRNA into the cytoplasm by liposomal bubbles and ultrasound","volume":"132","author":"Negishi","year":"2008","journal-title":"J. Control. Release"},{"key":"ref_198","doi-asserted-by":"crossref","first-page":"159","DOI":"10.1016\/j.ejps.2010.03.019","article-title":"Gene delivery by lipoplexes and polyplexes","volume":"40","author":"Sun","year":"2010","journal-title":"Eur. J. Pharm. Sci."},{"key":"ref_199","doi-asserted-by":"crossref","first-page":"146","DOI":"10.1089\/nat.2018.0721","article-title":"Lipid nanoparticles enabling gene therapies: From concepts to clinical utility","volume":"28","author":"Kulkarni","year":"2018","journal-title":"Nucleic Acid Ther."},{"key":"ref_200","doi-asserted-by":"crossref","first-page":"606","DOI":"10.1021\/acs.biomac.7b01686","article-title":"Highly stable RNA capture by dense cationic polymer brushes for the design of cytocompatible, serum-stable siRNA delivery vectors","volume":"19","author":"Li","year":"2018","journal-title":"Biomacromolecules"},{"key":"ref_201","doi-asserted-by":"crossref","first-page":"1377","DOI":"10.1016\/j.nano.2016.12.014","article-title":"Design of lipid nanoparticles for in vitro and in vivo delivery of plasmid DNA","volume":"13","author":"Kulkarni","year":"2017","journal-title":"Nanomedicine"},{"key":"ref_202","doi-asserted-by":"crossref","first-page":"1651","DOI":"10.1016\/j.addr.2003.08.005","article-title":"Atelocollagen for protein and gene delivery","volume":"55","author":"Sano","year":"2003","journal-title":"Adv. Drug Deliv. Rev."},{"key":"ref_203","doi-asserted-by":"crossref","first-page":"31","DOI":"10.2174\/1566523013348887","article-title":"Biomaterials for gene delivery: Atelocollagen-mediated controlled release of molecular medicines","volume":"1","author":"Ochiya","year":"2001","journal-title":"Curr. Gene Ther."},{"key":"ref_204","doi-asserted-by":"crossref","first-page":"9480","DOI":"10.1021\/ja016062m","article-title":"A novel biodegradable gene carrier based on polyphosphoester","volume":"123","author":"Wang","year":"2001","journal-title":"J. Am. Chem. Soc."},{"key":"ref_205","doi-asserted-by":"crossref","first-page":"4495","DOI":"10.1016\/S0142-9612(03)00347-8","article-title":"Polyion complex micelles from plasmid DNA and poly(ethylene glycol)-poly(L-lysine) block copolymer as serum-tolerable polyplex system: Physicochemical properties of micelles relevant to gene transfection efficiency","volume":"24","author":"Itaka","year":"2003","journal-title":"Biomaterials"},{"key":"ref_206","doi-asserted-by":"crossref","first-page":"2076","DOI":"10.1016\/j.drudis.2019.06.014","article-title":"Drug repurposing: A promising tool to accelerate the drug discovery process","volume":"24","author":"Parvathaneni","year":"2019","journal-title":"Drug Discov. Today"},{"key":"ref_207","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1016\/j.actbio.2017.12.023","article-title":"Repurposing disulfiram for cancer therapy via targeted nanotechnology through enhanced tumor mass penetration and disassembly","volume":"68","author":"He","year":"2018","journal-title":"Acta Biomater."},{"key":"ref_208","doi-asserted-by":"crossref","first-page":"399","DOI":"10.1093\/jnci\/djy131","article-title":"Combining tumor microenvironment modulating nanoparticles with doxorubicin to enhance chemotherapeutic efficacy and boost antitumor immunity","volume":"111","author":"Amini","year":"2019","journal-title":"J. Natl. Cancer Inst."},{"key":"ref_209","doi-asserted-by":"crossref","first-page":"7906","DOI":"10.7150\/thno.38425","article-title":"Recent advances and challenges of repurposing nanoparticle-based drug delivery systems to enhance cancer immunotherapy","volume":"9","author":"Lim","year":"2019","journal-title":"Theranostics"},{"key":"ref_210","doi-asserted-by":"crossref","first-page":"113","DOI":"10.7150\/ntno.51955","article-title":"Resveratrol loaded polymeric micelles for theranostic targeting of breast cancer cells","volume":"5","author":"Gregoriou","year":"2021","journal-title":"Nanotheranostics"},{"key":"ref_211","doi-asserted-by":"crossref","first-page":"624","DOI":"10.1002\/mus.10341","article-title":"Gentamicin fails to increase dystrophin expression in dystrophin-deficient muscle","volume":"27","author":"Dunant","year":"2003","journal-title":"Muscle Nerve"},{"key":"ref_212","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1016\/j.semcdb.2017.11.011","article-title":"Function of the myogenic regulatory factors Myf5, MyoD, Myogenin and MRF4 in skeletal muscle, satellite cells and regenerative myogenesis","volume":"72","author":"Zammit","year":"2017","journal-title":"Semin. Cell Dev. Biol."},{"key":"ref_213","doi-asserted-by":"crossref","first-page":"7S","DOI":"10.1016\/S0041-1345(03)00211-2","article-title":"Sirolimus: Its discovery, biological properties, and mechanism of action","volume":"35","author":"Sehgal","year":"2003","journal-title":"Transplant. Proc."},{"key":"ref_214","doi-asserted-by":"crossref","first-page":"1043","DOI":"10.3109\/10731199409138801","article-title":"Pharmacokinetics and side effects of perfluorocarbon-based blood substitutes","volume":"22","author":"Flaim","year":"1994","journal-title":"Artif. Cells. Blood Substit. Immobil. Biotechnol."},{"key":"ref_215","doi-asserted-by":"crossref","first-page":"R93","DOI":"10.1186\/cc364","article-title":"Blood substitutes. Artificial oxygen carriers: Perfluorocarbon emulsions","volume":"3","author":"Spahn","year":"1999","journal-title":"Crit. Care"},{"key":"ref_216","doi-asserted-by":"crossref","first-page":"118","DOI":"10.1016\/j.ijpharm.2016.05.043","article-title":"Effect of particle size on their accumulation in an inflammatory lesion in a dextran sulfate sodium (DSS)-induced colitis model","volume":"509","author":"Watanabe","year":"2016","journal-title":"Int. J. Pharm."},{"key":"ref_217","doi-asserted-by":"crossref","first-page":"15863","DOI":"10.1039\/C5NR03626G","article-title":"Nanoparticle distribution during systemic inflammation is size-dependent and organ-specific","volume":"7","author":"Chen","year":"2015","journal-title":"Nanoscale"},{"key":"ref_218","doi-asserted-by":"crossref","first-page":"816","DOI":"10.1126\/science.1225829","article-title":"A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity","volume":"337","author":"Jinek","year":"2012","journal-title":"Science"},{"key":"ref_219","doi-asserted-by":"crossref","first-page":"905","DOI":"10.1080\/17425247.2018.1517746","article-title":"Delivery approaches for CRISPR\/Cas9 therapeutics in vivo: Advances and challenges","volume":"15","author":"Luther","year":"2018","journal-title":"Expert Opin. Drug Deliv."},{"key":"ref_220","doi-asserted-by":"crossref","first-page":"1234","DOI":"10.1080\/10717544.2018.1474964","article-title":"Delivering CRISPR: A review of the challenges and approaches","volume":"25","author":"Lino","year":"2018","journal-title":"Drug Deliv."},{"key":"ref_221","doi-asserted-by":"crossref","first-page":"1075","DOI":"10.1038\/mt.2014.30","article-title":"Gold nanoparticles for nucleic acid delivery","volume":"22","author":"Ding","year":"2014","journal-title":"Mol. Ther."},{"key":"ref_222","doi-asserted-by":"crossref","first-page":"662","DOI":"10.1021\/nl052396o","article-title":"Determining the size and shape dependence of gold nanoparticle uptake into mammalian cells","volume":"6","author":"Chithrani","year":"2006","journal-title":"Nano Lett."},{"key":"ref_223","first-page":"487","article-title":"Immortalized human myotonic dystrophy muscle cell lines to assess therapeutic compounds","volume":"10","author":"Arandel","year":"2017","journal-title":"Dis. Model. Mech."},{"key":"ref_224","doi-asserted-by":"crossref","first-page":"361","DOI":"10.3389\/fneur.2018.00361","article-title":"Cells of matter\u2014in vitro models for myotonic dystrophy","volume":"9","author":"Matloka","year":"2018","journal-title":"Front. Neurol."},{"key":"ref_225","doi-asserted-by":"crossref","first-page":"1435","DOI":"10.2353\/ajpath.2009.080560","article-title":"Large CTG repeats trigger P16-dependent premature senescence in myotonic dystrophy type 1 muscle precursor cells","volume":"174","author":"Bigot","year":"2009","journal-title":"Am. J. Pathol."},{"key":"ref_226","doi-asserted-by":"crossref","first-page":"614","DOI":"10.1016\/0014-4827(65)90211-9","article-title":"The limited in vitro lifetime of human diploid cell strains","volume":"37","author":"Hayflick","year":"1965","journal-title":"Exp. Cell Res."},{"key":"ref_227","doi-asserted-by":"crossref","first-page":"711","DOI":"10.1016\/S0531-5565(00)00151-0","article-title":"Skeletal muscle regeneration and the mitotic clock","volume":"35","author":"Renault","year":"2000","journal-title":"Exp. Gerontol."},{"key":"ref_228","first-page":"2444","article-title":"Premature senescence in primary muscle cultures of myotonic dystrophy type 2 is not associated with P16 induction","volume":"58","author":"Renna","year":"2014","journal-title":"Eur. J. Histochem."},{"key":"ref_229","doi-asserted-by":"crossref","first-page":"603","DOI":"10.1111\/j.1365-2990.2009.01014.x","article-title":"Satellite cell dysfunction contributes to the progressive muscle atrophy in myotonic dystrophy type 1","volume":"35","author":"Thornell","year":"2009","journal-title":"Neuropathol. Appl. Neurobiol."},{"key":"ref_230","first-page":"54078","article-title":"Modeling myotonic dystrophy 1 in C2C12 myoblast cells","volume":"113","author":"Liang","year":"2016","journal-title":"J. Vis. Exp."},{"key":"ref_231","doi-asserted-by":"crossref","first-page":"34","DOI":"10.1186\/2044-5040-1-34","article-title":"Immortalized pathological human myoblasts: Towards a universal tool for the study of neuromuscular disorders","volume":"1","author":"Mamchaoui","year":"2011","journal-title":"Skelet. Muscle"},{"key":"ref_232","doi-asserted-by":"crossref","first-page":"1130","DOI":"10.1002\/btpr.223","article-title":"Differentiation rather than aging of muscle stem cells abolishes their telomerase activity","volume":"25","author":"Carlson","year":"2009","journal-title":"Biotechnol. Prog."},{"key":"ref_233","doi-asserted-by":"crossref","unstructured":"Massenet, J., Gitiaux, C., Magnan, M., Cuvellier, S., Hubas, A., Nusbaum, P., Dilworth, F.J., Desguerre, I., and Chazaud, B. (2020). Derivation and characterization of immortalized human muscle satellite cell clones from muscular dystrophy patients and healthy individuals. Cells, 9.","DOI":"10.3390\/cells9081780"},{"key":"ref_234","doi-asserted-by":"crossref","first-page":"784","DOI":"10.1089\/hum.2008.163","article-title":"Immortalized skin fibroblasts expressing conditional MyoD as a renewable and reliable source of converted human muscle cells to assess therapeutic strategies for muscular mystrophies: Validation of an exon-skipping approach to restore dystrophin in Duchenne muscular dystrophy Cells","volume":"20","author":"Chaouch","year":"2009","journal-title":"Hum. Gene Ther."},{"key":"ref_235","doi-asserted-by":"crossref","first-page":"276","DOI":"10.1016\/j.nmd.2006.12.010","article-title":"Dystrophinopathy carrier determination and detection of protein deficiencies in muscular dystrophy using lentiviral MyoD-forced myogenesis","volume":"17","author":"Cooper","year":"2007","journal-title":"Neuromuscul. Disord."},{"key":"ref_236","doi-asserted-by":"crossref","first-page":"207","DOI":"10.1089\/adt.2014.573","article-title":"Three-dimensional cell culture systems and their applications in drug discovery and cell-based biosensors","volume":"12","author":"Edmondson","year":"2014","journal-title":"Assay Drug Dev. Technol."},{"key":"ref_237","doi-asserted-by":"crossref","first-page":"266","DOI":"10.1152\/physiol.00036.2016","article-title":"Modeling physiological events in 2D vs. 3D cell culture","volume":"32","author":"Duval","year":"2017","journal-title":"Physiology"},{"key":"ref_238","doi-asserted-by":"crossref","first-page":"746","DOI":"10.1111\/joa.12257","article-title":"Advances in 3D cell culture technologies enabling tissue-like structures to be created in vitro","volume":"227","author":"Knight","year":"2015","journal-title":"J. Anat."},{"key":"ref_239","doi-asserted-by":"crossref","first-page":"19529","DOI":"10.1038\/s41598-020-74191-w","article-title":"3D micro-organisation printing of mammalian cells to generate biological tissues","volume":"10","author":"Jeffries","year":"2020","journal-title":"Sci. Rep."},{"key":"ref_240","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1038\/s41419-019-1647-5","article-title":"Dissecting cell diversity and connectivity in skeletal muscle for myogenesis","volume":"10","author":"Liu","year":"2019","journal-title":"Cell Death Dis."},{"key":"ref_241","doi-asserted-by":"crossref","first-page":"5508","DOI":"10.1073\/pnas.1402723111","article-title":"Biomimetic Engineered muscle with capacity for vascular integration and functional maturation in vivo","volume":"111","author":"Juhas","year":"2014","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_242","doi-asserted-by":"crossref","first-page":"8905","DOI":"10.1016\/j.biomaterials.2011.08.019","article-title":"The role of multifunctional delivery scaffold in the ability of cultured myoblasts to promote muscle regeneration","volume":"32","author":"Borselli","year":"2011","journal-title":"Biomaterials"},{"key":"ref_243","doi-asserted-by":"crossref","first-page":"1399","DOI":"10.1039\/C8LC00241J","article-title":"Powering ex vivo tissue models in microfluidic systems","volume":"18","author":"McLean","year":"2018","journal-title":"Lab. Chip"},{"key":"ref_244","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1016\/bs.pbr.2016.11.003","article-title":"Ex vivo gene therapy for the treatment of neurological disorders","volume":"230","author":"Gowing","year":"2017","journal-title":"Prog. Brain Res."},{"key":"ref_245","doi-asserted-by":"crossref","first-page":"146","DOI":"10.1016\/j.biomaterials.2018.05.012","article-title":"Advances in ex vivo models and lab-on-a-chip devices for neural tissue engineering","volume":"198","author":"Mobini","year":"2019","journal-title":"Biomaterials"},{"key":"ref_246","doi-asserted-by":"crossref","first-page":"248","DOI":"10.1080\/03008207.2019.1662409","article-title":"Skeletal muscle explants: Ex-vivo models to study cellular behavior in a complex tissue environment","volume":"61","author":"Smith","year":"2020","journal-title":"Connect. Tissue Res."},{"key":"ref_247","doi-asserted-by":"crossref","first-page":"1165","DOI":"10.1021\/ar4002608","article-title":"Microfluidics-based in vivo mimetic systems for the study of cellular biology","volume":"47","author":"Kim","year":"2014","journal-title":"Acc. Chem. Res."},{"key":"ref_248","first-page":"2862","article-title":"Incubation under fluid dynamic conditions markedly improves the structural preservation in vitro of explanted skeletal muscles","volume":"61","author":"Carton","year":"2017","journal-title":"Eur. J. Histochem."},{"key":"ref_249","doi-asserted-by":"crossref","first-page":"1420","DOI":"10.1038\/srep01420","article-title":"Generation of ex vivo-vascularized muscle engineered tissue (X-MET)","volume":"3","author":"Carosio","year":"2013","journal-title":"Sci. Rep."},{"key":"ref_250","doi-asserted-by":"crossref","first-page":"565","DOI":"10.1016\/j.copbio.2007.11.008","article-title":"Nanotoxicity: The growing need for in vivo study","volume":"18","author":"Fischer","year":"2007","journal-title":"Curr. Opin. Biotechnol."},{"key":"ref_251","doi-asserted-by":"crossref","first-page":"S63","DOI":"10.5435\/00124635-200800001-00013","article-title":"What experimental approaches (eg, in vivo, in vitro, tissue retrieval) are effective in investigating the biologic effects of particles?","volume":"16","author":"Bostrom","year":"2008","journal-title":"J. Am. Acad. Orthop. Surg."},{"key":"ref_252","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1242\/dmm.018424","article-title":"Animal models of Duchenne muscular dystrophy: From basic mechanisms to gene therapy","volume":"8","author":"McGreevy","year":"2015","journal-title":"Dis. Model. Mech."},{"key":"ref_253","doi-asserted-by":"crossref","first-page":"dmm035774","DOI":"10.1242\/dmm.035774","article-title":"Tracking progress: An update on animal models for Duchenne muscular dystrophy","volume":"11","author":"Wells","year":"2018","journal-title":"Dis. Model. Mech."},{"key":"ref_254","doi-asserted-by":"crossref","first-page":"165","DOI":"10.1046\/j.1365-2613.2003.00354.x","article-title":"Duchenne\u2019s muscular dystrophy: Animal models used to investigate pathogenesis and develop therapeutic strategies","volume":"84","author":"Collins","year":"2003","journal-title":"Int. J. Exp. Pathol."},{"key":"ref_255","doi-asserted-by":"crossref","first-page":"1189","DOI":"10.1073\/pnas.81.4.1189","article-title":"X Chromosome-linked muscular dystrophy (mdx) in the mouse","volume":"81","author":"Bulfield","year":"1984","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_256","doi-asserted-by":"crossref","first-page":"8110","DOI":"10.1096\/fj.201802488R","article-title":"Natural disease history of the D2-mdx mouse model for Duchenne muscular dystrophy","volume":"33","author":"Putker","year":"2019","journal-title":"FASEB J."},{"key":"ref_257","doi-asserted-by":"crossref","first-page":"803","DOI":"10.1002\/mus.23341","article-title":"A new model of experimental fibrosis in hindlimb skeletal muscle of adult mdx mouse mimicking muscular dystrophy","volume":"45","author":"Desguerre","year":"2012","journal-title":"Muscle Nerve"},{"key":"ref_258","doi-asserted-by":"crossref","first-page":"83","DOI":"10.1016\/B978-0-12-394596-9.00004-4","article-title":"Animal models of muscular dystrophy","volume":"105","author":"Ng","year":"2012","journal-title":"Prog. Mol. Biol. Transl. Sci."},{"key":"ref_259","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1186\/s13395-017-0124-z","article-title":"The golden retriever model of Duchenne muscular dystrophy","volume":"7","author":"Kornegay","year":"2017","journal-title":"Skelet. Muscle"},{"key":"ref_260","doi-asserted-by":"crossref","first-page":"5331","DOI":"10.1073\/pnas.1102116108","article-title":"Drug screening in a zebrafish model of Duchenne muscular dystrophy","volume":"108","author":"Kawahara","year":"2011","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_261","doi-asserted-by":"crossref","first-page":"271","DOI":"10.3233\/JND-190389","article-title":"Discovery of novel therapeutics for muscular dystrophies using zebrafish phenotypic screens","volume":"6","author":"Widrick","year":"2019","journal-title":"J. Neuromuscul. Dis."},{"key":"ref_262","doi-asserted-by":"crossref","first-page":"1390","DOI":"10.1016\/j.bbadis.2013.03.002","article-title":"RNA toxicity in human disease and animal models: From the uncovering of a new mechanism to the development of promising therapies","volume":"1832","author":"Sicot","year":"2013","journal-title":"Biochim. Biophys. Acta"},{"key":"ref_263","doi-asserted-by":"crossref","first-page":"506","DOI":"10.1016\/j.molmed.2011.05.004","article-title":"Myotonic dystrophy mouse models: Towards rational therapy development","volume":"17","author":"Cooper","year":"2011","journal-title":"Trends Mol. Med."},{"key":"ref_264","doi-asserted-by":"crossref","first-page":"1183","DOI":"10.1016\/j.bbadis.2007.08.004","article-title":"DM1 CTG expansions affect insulin receptor isoforms expression in various tissues of transgenic mice","volume":"1772","author":"Huguet","year":"2007","journal-title":"Biochim. Biophys. Acta"},{"key":"ref_265","doi-asserted-by":"crossref","first-page":"2087","DOI":"10.1093\/hmg\/ddl132","article-title":"Failure of MBNL1-dependent post-natal splicing transitions in myotonic dystrophy","volume":"15","author":"Lin","year":"2006","journal-title":"Hum. Mol. Genet."},{"key":"ref_266","doi-asserted-by":"crossref","first-page":"1769","DOI":"10.1126\/science.289.5485.1769","article-title":"Myotonic dystrophy in transgenic mice expressing an expanded CUG repeat","volume":"289","author":"Mankodi","year":"2000","journal-title":"Science"},{"key":"ref_267","doi-asserted-by":"crossref","first-page":"35","DOI":"10.1016\/S1097-2765(02)00563-4","article-title":"Expanded CUG repeats trigger aberrant splicing of ClC-1 chloride channel pre-mRNA and hyperexcitability of skeletal muscle in myotonic dystrophy","volume":"10","author":"Mankodi","year":"2002","journal-title":"Mol. Cell"},{"key":"ref_268","doi-asserted-by":"crossref","first-page":"2717","DOI":"10.1093\/hmg\/10.23.2717","article-title":"Mice transgenic for the human myotonic dystrophy region with expanded CTG repeats display muscular and brain abnormalities","volume":"10","author":"Seznec","year":"2001","journal-title":"Hum. Mol. Genet."},{"key":"ref_269","doi-asserted-by":"crossref","unstructured":"Hinman, M.N., Richardson, J.I., Sockol, R.A., Aronson, E.D., Stednitz, S.J., Murray, K.N., Berglund, J.A., and Guillemin, K. (2020). Zebrafish mbnl mutants model physical and molecular phenotypes of myotonic dystrophy. bioRxiv, 665380.","DOI":"10.1101\/665380"},{"key":"ref_270","doi-asserted-by":"crossref","first-page":"381","DOI":"10.1242\/dmm.004150","article-title":"Zebrafish deficient for Muscleblind-like 2 exhibit features of myotonic dystrophy","volume":"4","author":"Buxton","year":"2011","journal-title":"Dis. Model. Mech."},{"key":"ref_271","doi-asserted-by":"crossref","first-page":"118526","DOI":"10.1016\/j.ijpharm.2019.118526","article-title":"Rationally designed hyaluronic acid-based nano-complexes for pentamidine delivery","volume":"568","author":"Carton","year":"2019","journal-title":"Int. J. Pharm."},{"key":"ref_272","doi-asserted-by":"crossref","first-page":"1297","DOI":"10.1016\/j.xphs.2019.11.011","article-title":"Pentamidine-loaded lipid and polymer nanocarriers as tunable anticancer drug delivery systems","volume":"109","author":"Stella","year":"2020","journal-title":"J. Pharm. Sci."},{"key":"ref_273","doi-asserted-by":"crossref","first-page":"927","DOI":"10.1089\/hum.2014.073","article-title":"Biodistribution studies of polymeric nanoparticles for drug delivery in mice","volume":"25","author":"Falzarano","year":"2014","journal-title":"Hum Gene Ther."},{"key":"ref_274","doi-asserted-by":"crossref","first-page":"196","DOI":"10.1016\/j.actbio.2020.10.009","article-title":"Polymeric nanoparticles functionalized with muscle-homing peptides for targeted delivery of phosphatase and tensin homolog inhibitor to skeletal muscle","volume":"118","author":"Huang","year":"2020","journal-title":"Acta Biomater."},{"key":"ref_275","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1038\/sj.gt.3302891","article-title":"Gene therapy progress and prospects: Hydrodynamic gene delivery","volume":"14","author":"Herweijer","year":"2007","journal-title":"Gene Ther."},{"key":"ref_276","doi-asserted-by":"crossref","first-page":"755","DOI":"10.1016\/j.gie.2018.06.022","article-title":"Successful liver-directed gene delivery by ERCP-guided hydrodynamic injection (with Videos)","volume":"88","author":"Kumbhari","year":"2018","journal-title":"Gastrointest. Endosc."},{"key":"ref_277","doi-asserted-by":"crossref","first-page":"e3150","DOI":"10.1002\/jgm.3150","article-title":"Gene transfer to skeletal muscle using hydrodynamic limb vein injection: Current applications, hurdles and possible optimizations","volume":"22","author":"Midoux","year":"2020","journal-title":"J. Gene Med."},{"key":"ref_278","doi-asserted-by":"crossref","first-page":"317","DOI":"10.1615\/CritRevTherDrugCarrierSyst.2017017845","article-title":"Recent advances in nanoparticle-based targeted drug-delivery systems against cancer and role of tumor microenvironment","volume":"34","author":"Ashfaq","year":"2017","journal-title":"Crit. Rev. Ther. Drug Carrier Syst."},{"key":"ref_279","doi-asserted-by":"crossref","first-page":"1873","DOI":"10.2217\/nnm-2017-0043","article-title":"Therapeutic targeting in nanomedicine: The future lies in recombinant antibodies","volume":"12","author":"Pietersz","year":"2017","journal-title":"Nanomedicine"},{"key":"ref_280","doi-asserted-by":"crossref","first-page":"861","DOI":"10.1038\/333861a0","article-title":"Immunostaining of skeletal and cardiac muscle surface membrane with antibody against Duchenne muscular dystrophy peptide","volume":"333","author":"Arahata","year":"1988","journal-title":"Nature"},{"key":"ref_281","doi-asserted-by":"crossref","first-page":"570","DOI":"10.1038\/s41586-018-0121-3","article-title":"Mxra8 is a receptor for multiple arthritogenic alphaviruses","volume":"557","author":"Zhang","year":"2018","journal-title":"Nature"},{"key":"ref_282","doi-asserted-by":"crossref","first-page":"285101","DOI":"10.1088\/0957-4484\/26\/28\/285101","article-title":"Targeting B16 tumors in vivo with peptide-conjugated gold nanoparticles","volume":"26","author":"Poon","year":"2015","journal-title":"Nanotechnology"},{"key":"ref_283","doi-asserted-by":"crossref","first-page":"21881","DOI":"10.1038\/srep21881","article-title":"Receptor-targeted liposome-peptide-siRNA nanoparticles represent an efficient delivery system for MRTF silencing in conjunctival fibrosis","volume":"6","author":"Tagalakis","year":"2016","journal-title":"Sci. Rep."},{"key":"ref_284","doi-asserted-by":"crossref","first-page":"21704","DOI":"10.1038\/s41598-020-78601-x","article-title":"Surface functionalisation of poly-APO- b -polyol ester cross-linked copolymers as core\u2013shell nanoparticles for targeted breast cancer therapy","volume":"10","author":"Tajau","year":"2020","journal-title":"Sci. Rep."},{"key":"ref_285","doi-asserted-by":"crossref","first-page":"953","DOI":"10.1038\/gt.2009.59","article-title":"A muscle-targeting peptide displayed on AAV2 improves muscle tropism on systemic delivery","volume":"16","author":"Yu","year":"2009","journal-title":"Gene Ther."},{"key":"ref_286","doi-asserted-by":"crossref","first-page":"1873","DOI":"10.1016\/j.peptides.2010.06.036","article-title":"Identification of a novel muscle targeting peptide in mdx mice","volume":"31","author":"Seow","year":"2010","journal-title":"Peptides"},{"key":"ref_287","doi-asserted-by":"crossref","first-page":"630","DOI":"10.1016\/j.ebiom.2019.06.036","article-title":"Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases","volume":"45","author":"Tsoumpra","year":"2019","journal-title":"EBioMedicine"},{"key":"ref_288","doi-asserted-by":"crossref","first-page":"1333","DOI":"10.1038\/mt.2014.63","article-title":"Effective dystrophin restoration by a novel muscle-homing peptide-morpholino conjugate in dystrophin-deficient mdx mice","volume":"22","author":"Gao","year":"2014","journal-title":"Mol. Ther."}],"container-title":["Pharmaceutics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/1999-4923\/13\/2\/278\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,11]],"date-time":"2025-10-11T05:26:11Z","timestamp":1760160371000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/1999-4923\/13\/2\/278"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,2,19]]},"references-count":288,"journal-issue":{"issue":"2","published-online":{"date-parts":[[2021,2]]}},"alternative-id":["pharmaceutics13020278"],"URL":"https:\/\/doi.org\/10.3390\/pharmaceutics13020278","relation":{},"ISSN":["1999-4923"],"issn-type":[{"value":"1999-4923","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,2,19]]}}}