{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,2]],"date-time":"2026-07-02T20:17:08Z","timestamp":1783023428765,"version":"3.54.6"},"reference-count":143,"publisher":"Elsevier BV","issue":"11","license":[{"start":{"date-parts":[[2024,11,1]],"date-time":"2024-11-01T00:00:00Z","timestamp":1730419200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,11,1]],"date-time":"2024-11-01T00:00:00Z","timestamp":1730419200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,8,23]],"date-time":"2024-08-23T00:00:00Z","timestamp":1724371200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100020668","name":"European Research Executive Agency","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100020668","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004243","name":"Prinses Beatrix Spierfonds","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004243","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010665","name":"H2020 Marie Sk\u0142odowska-Curie Actions","doi-asserted-by":"publisher","award":["101072427"],"award-info":[{"award-number":["101072427"]}],"id":[{"id":"10.13039\/100010665","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003246","name":"Nederlandse Organisatie voor Wetenschappelijk Onderzoek","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003246","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000780","name":"European Union","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000780","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100007423","name":"Duchenne Parent Project","doi-asserted-by":"publisher","award":["22.012"],"award-info":[{"award-number":["22.012"]}],"id":[{"id":"10.13039\/501100007423","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2024,11]]},"DOI":"10.1016\/j.ymthe.2024.08.016","type":"journal-article","created":{"date-parts":[[2024,8,22]],"date-time":"2024-08-22T21:41:34Z","timestamp":1724362894000},"page":"3793-3807","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":7,"title":["On RNA-programmable gene modulation as a versatile set of principles targeting muscular dystrophies"],"prefix":"10.1016","volume":"32","author":[{"given":"Sabrina","family":"Capelletti","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sof\u00eda C.","family":"Garc\u00eda Soto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8111-1073","authenticated-orcid":false,"given":"Manuel A.F.V.","family":"Gon\u00e7alves","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2024.08.016_bib1","doi-asserted-by":"crossref","first-page":"3827","DOI":"10.1038\/onc.2011.552","article-title":"Targeting the epigenome for treatment of cancer","volume":"31","author":"Geutjes","year":"2012","journal-title":"Oncogene"},{"key":"10.1016\/j.ymthe.2024.08.016_bib2","doi-asserted-by":"crossref","first-page":"104094","DOI":"10.1016\/j.drudis.2024.104094","article-title":"Targeting histone deacetylases: Emerging applications beyond cancer","volume":"29","author":"Raouf","year":"2024","journal-title":"Drug Discov. Today"},{"key":"10.1016\/j.ymthe.2024.08.016_bib3","doi-asserted-by":"crossref","first-page":"643","DOI":"10.1016\/j.nmd.2016.07.002","article-title":"Histological effects of givinostat in boys with Duchenne muscular dystrophy","volume":"26","author":"Bettica","year":"2016","journal-title":"Neuromuscul. Disord."},{"key":"10.1016\/j.ymthe.2024.08.016_bib4","doi-asserted-by":"crossref","first-page":"4306","DOI":"10.3390\/ijms24054306","article-title":"Histone deacetylases: molecular mechanisms and therapeutic implications for muscular dystrophies","volume":"24","author":"Sandon\u00e0","year":"2023","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.ymthe.2024.08.016_bib5","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"},{"key":"10.1016\/j.ymthe.2024.08.016_bib6","doi-asserted-by":"crossref","first-page":"737","DOI":"10.1042\/bj20021321","article-title":"Histone deacetylases (HDACs): characterization of the classical HDAC family","volume":"370","author":"de Ruijter","year":"2003","journal-title":"Biochem. J."},{"key":"10.1016\/j.ymthe.2024.08.016_bib7","doi-asserted-by":"crossref","first-page":"278","DOI":"10.1016\/j.molmed.2024.01.007","article-title":"HDAC inhibitors as pharmacological treatment for Duchenne muscular dystrophy: a discovery journey from bench to patients","volume":"30","author":"Mozzetta","year":"2024","journal-title":"Trends Mol. Med."},{"key":"10.1016\/j.ymthe.2024.08.016_bib8","doi-asserted-by":"crossref","first-page":"19183","DOI":"10.1073\/pnas.0805514105","article-title":"HDAC2 blockade by nitric oxide and histone deacetylase inhibitors reveals a common target in Duchenne muscular dystrophy treatment","volume":"105","author":"Colussi","year":"2008","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2024.08.016_bib9","first-page":"385","article-title":"MAPK signaling pathways and HDAC3 activity are disrupted during differentiation of emerin-null myogenic progenitor cells","volume":"10","author":"Collins","year":"2017","journal-title":"Dis. Model. Mech."},{"key":"10.1016\/j.ymthe.2024.08.016_bib10","doi-asserted-by":"crossref","first-page":"1147","DOI":"10.1038\/nm1479","article-title":"Functional and morphological recovery of dystrophic muscles in mice treated with deacetylase inhibitors","volume":"12","author":"Minetti","year":"2006","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2024.08.016_bib11","doi-asserted-by":"crossref","first-page":"79","DOI":"10.2119\/molmed.2013.00011","article-title":"Preclinical studies in the mdx mouse model of duchenne muscular dystrophy with the histone deacetylase inhibitor givinostat","volume":"19","author":"Consalvi","year":"2013","journal-title":"Mol. Med."},{"key":"10.1016\/j.ymthe.2024.08.016_bib12","doi-asserted-by":"crossref","first-page":"856804","DOI":"10.3389\/fphar.2022.856804","article-title":"Dual blockade of misfolded alpha-sarcoglycan degradation by bortezomib and givinostat combination","volume":"13","author":"Hoch","year":"2022","journal-title":"Front. Pharmacol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib13","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":"10.1016\/j.ymthe.2024.08.016_bib14","doi-asserted-by":"crossref","first-page":"116481","DOI":"10.1016\/j.biopha.2024.116481","article-title":"Research progress on Sirtuins (SIRTs) family modulators","volume":"174","author":"Chen","year":"2024","journal-title":"Biomed. Pharmacother."},{"key":"10.1016\/j.ymthe.2024.08.016_bib15","doi-asserted-by":"crossref","first-page":"e1004490","DOI":"10.1371\/journal.pgen.1004490","article-title":"Muscle-specific SIRT1 gain-of-function increases slow-twitch fibers and ameliorates pathophysiology in a mouse model of Duchenne muscular dystrophy","volume":"10","author":"Chalkiadaki","year":"2014","journal-title":"PLoS Genet."},{"key":"10.1016\/j.ymthe.2024.08.016_bib16","doi-asserted-by":"crossref","first-page":"20585","DOI":"10.1038\/s41598-020-77197-6","article-title":"Resveratrol improves motor function in patients with muscular dystrophies: an open-label, single-arm, phase IIa study","volume":"10","author":"Kawamura","year":"2020","journal-title":"Sci. Rep."},{"key":"10.1016\/j.ymthe.2024.08.016_bib17","doi-asserted-by":"crossref","first-page":"393","DOI":"10.1016\/S1474-4422(24)00036-X","article-title":"Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial","volume":"23","author":"Mercuri","year":"2024","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib18","doi-asserted-by":"crossref","first-page":"1076","DOI":"10.1016\/j.cell.2024.01.042","article-title":"Past, present, and future of CRISPR genome editing technologies","volume":"187","author":"Pacesa","year":"2024","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2024.08.016_bib19","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1038\/s41580-023-00697-6","article-title":"CRISPR technologies for genome, epigenome and transcriptome editing","volume":"25","author":"Villiger","year":"2024","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib20","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":"10.1016\/j.ymthe.2024.08.016_bib21","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1016\/j.exphem.2022.03.002","article-title":"CRISPR-Cas9 gene editing induced complex on-target outcomes in human cells","volume":"110","author":"Wen","year":"2022","journal-title":"Exp. Hematol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib22","doi-asserted-by":"crossref","first-page":"247","DOI":"10.1016\/j.isci.2018.08.001","article-title":"DNA, RNA, and protein tools for editing the genetic information in human cells","volume":"6","author":"Chen","year":"2018","journal-title":"iScience"},{"key":"10.1016\/j.ymthe.2024.08.016_bib23","doi-asserted-by":"crossref","first-page":"974","DOI":"10.1093\/nar\/gkz1121","article-title":"Expanding the editable genome and CRISPR-Cas9 versatility using DNA cutting-free gene targeting based on in trans paired nicking","volume":"48","author":"Chen","year":"2020","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.ymthe.2024.08.016_bib24","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1038\/mt.2015.164","article-title":"Engineered viruses as genome editing devices","volume":"24","author":"Chen","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2024.08.016_bib25","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1186\/s13073-016-0316-x","article-title":"The emerging role of viral vectors as vehicles for DMD gene editing","volume":"23","author":"Maggio","year":"2016","journal-title":"Genome Med."},{"key":"10.1016\/j.ymthe.2024.08.016_bib26","doi-asserted-by":"crossref","first-page":"4439","DOI":"10.1038\/s41467-019-12449-2","article-title":"High levels of AAV vector integration into CRISPR-induced DNA breaks","volume":"10","author":"Hanlon","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2024.08.016_bib27","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1038\/s41591-019-0344-3","article-title":"Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy","volume":"25","author":"Nelson","year":"2019","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2024.08.016_bib28","doi-asserted-by":"crossref","first-page":"657","DOI":"10.1038\/s41467-017-00687-1","article-title":"In trans paired nicking triggers seamless genome editing without double-stranded DNA cutting","volume":"8","author":"Chen","year":"2017","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2024.08.016_bib29","doi-asserted-by":"crossref","first-page":"223","DOI":"10.1101\/gr.226027.117","article-title":"Precise and efficient nucleotide substitution near genomic nick via noncanonical homology-directed repair","volume":"28","author":"Nakajima","year":"2018","journal-title":"Genome Res."},{"key":"10.1016\/j.ymthe.2024.08.016_bib30","doi-asserted-by":"crossref","first-page":"3107","DOI":"10.1016\/j.ymthe.2021.09.002","article-title":"In\u00a0vivo somatic cell base editing and prime editing","volume":"29","author":"Newby","year":"2021","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2024.08.016_bib31","doi-asserted-by":"crossref","first-page":"1195","DOI":"10.1016\/j.celrep.2019.12.064","article-title":"Tandem paired nicking promotes precise genome editing with scarce interference by p53","volume":"30","author":"Hyodo","year":"2020","journal-title":"Cell Rep."},{"key":"10.1016\/j.ymthe.2024.08.016_bib32","doi-asserted-by":"crossref","first-page":"3465","DOI":"10.1093\/nar\/gkad165","article-title":"Precise homology-directed installation of large genomic edits in human cells with cleaving and nicking high-specificity Cas9 variants","volume":"51","author":"Wang","year":"2023","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.ymthe.2024.08.016_bib33","doi-asserted-by":"crossref","first-page":"1173","DOI":"10.1093\/nar\/gkaa1236","article-title":"Precise and broad scope genome editing based on high-specificity Cas9 nickases","volume":"49","author":"Wang","year":"2021","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.ymthe.2024.08.016_bib34","doi-asserted-by":"crossref","first-page":"877","DOI":"10.1038\/s41587-023-01915-4","article-title":"Genotoxic effects of base and prime editing in human hematopoietic stem cells","volume":"42","author":"Fiumara","year":"2024","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib35","doi-asserted-by":"crossref","first-page":"8241","DOI":"10.1073\/pnas.131009198","article-title":"Single-strand interruptions in replicating chromosomes cause double-strand breaks","volume":"98","author":"Kuzminov","year":"2001","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2024.08.016_bib36","doi-asserted-by":"crossref","first-page":"2212","DOI":"10.1038\/s41467-019-09983-4","article-title":"Development of hRad51-Cas9 nickase fusions that mediate HDR without double-stranded breaks","volume":"10","author":"Rees","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2024.08.016_bib37","doi-asserted-by":"crossref","first-page":"563","DOI":"10.1038\/335563a0","article-title":"GAL4-VP16 is an unusually potent transcriptional activator","volume":"335","author":"Sadowski","year":"1988","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2024.08.016_bib38","doi-asserted-by":"crossref","first-page":"977","DOI":"10.1038\/nmeth.2598","article-title":"CRISPR RNA-guided activation of endogenous human genes","volume":"10","author":"Maeder","year":"2013","journal-title":"Nat Methods"},{"key":"10.1016\/j.ymthe.2024.08.016_bib39","doi-asserted-by":"crossref","first-page":"462","DOI":"10.1016\/j.omtn.2022.07.013","article-title":"CRISPR-mediated activation of autism gene Itgb3 restores cortical network excitability via mGluR5 signaling","volume":"29","author":"Jaudon","year":"2022","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.ymthe.2024.08.016_bib40","doi-asserted-by":"crossref","first-page":"1777","DOI":"10.1016\/j.stemcr.2021.05.020","article-title":"Reprogramming astrocytes to motor neurons by activation of endogenous Ngn2 and Isl1","volume":"16","author":"Zhou","year":"2021","journal-title":"Stem Cell Rep."},{"key":"10.1016\/j.ymthe.2024.08.016_bib41","doi-asserted-by":"crossref","first-page":"891","DOI":"10.1093\/brain\/awaa045","article-title":"In\u00a0vivo CRISPRa decreases seizures and rescues cognitive deficits in a rodent model of epilepsy","volume":"143","author":"Colasante","year":"2020","journal-title":"Brain"},{"key":"10.1016\/j.ymthe.2024.08.016_bib42","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1016\/j.stemcr.2021.12.017","article-title":"CRISPR activation enables high-fidelity reprogramming into human pluripotent stem cells","volume":"17","author":"Sokka","year":"2022","journal-title":"Stem Cell Rep."},{"key":"10.1016\/j.ymthe.2024.08.016_bib43","doi-asserted-by":"crossref","first-page":"940","DOI":"10.1016\/j.stemcr.2014.09.013","article-title":"A CRISPR\/Cas9-based system for reprogramming cell lineage specification","volume":"3","author":"Chakraborty","year":"2014","journal-title":"Stem Cell Rep."},{"key":"10.1016\/j.ymthe.2024.08.016_bib44","doi-asserted-by":"crossref","first-page":"635","DOI":"10.1016\/j.cell.2014.09.039","article-title":"A protein-tagging system for signal amplification in gene expression and fluorescence imaging","volume":"159","author":"Tanenbaum","year":"2014","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2024.08.016_bib45","doi-asserted-by":"crossref","first-page":"170","DOI":"10.1038\/nbt.4062","article-title":"Dual gene activation and knockout screen reveals directional dependencies in genetic networks","volume":"36","author":"Boettcher","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib46","doi-asserted-by":"crossref","first-page":"758","DOI":"10.1016\/j.stem.2018.09.003","article-title":"CRISPR activation screens systematically identify factors that drive neuronal fate and reprogramming","volume":"23","author":"Liu","year":"2018","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.ymthe.2024.08.016_bib47","doi-asserted-by":"crossref","first-page":"2761","DOI":"10.1158\/0008-5472.CAN-21-4009","article-title":"A programmable in\u00a0vivo CRISPR activation model elucidates the oncogenic and immunosuppressive functions of MYC in lung adenocarcinoma","volume":"82","author":"Thege","year":"2022","journal-title":"Cancer Res."},{"key":"10.1016\/j.ymthe.2024.08.016_bib48","doi-asserted-by":"crossref","first-page":"1494","DOI":"10.1038\/s41590-019-0500-4","article-title":"Multiplexed activation of endogenous genes by CRISPRa elicits potent antitumor immunity","volume":"20","author":"Wang","year":"2019","journal-title":"Nat. Immunol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib49","doi-asserted-by":"crossref","first-page":"e14797","DOI":"10.15252\/emmm.202114797","article-title":"Parkinson\u2019s disease motor symptoms rescue by CRISPRa-reprogramming astrocytes into GABAergic neurons","volume":"14","author":"Giehrl-Schwab","year":"2022","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.ymthe.2024.08.016_bib50","doi-asserted-by":"crossref","first-page":"2770","DOI":"10.1038\/s41467-021-22932-4","article-title":"Tissue-specific activation of gene expression by the Synergistic Activation Mediator (SAM) CRISPRa system in mice","volume":"12","author":"Hunt","year":"2021","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2024.08.016_bib51","doi-asserted-by":"crossref","first-page":"326","DOI":"10.1038\/nmeth.3312","article-title":"Highly efficient Cas9-mediated transcriptional programming","volume":"12","author":"Chavez","year":"2015","journal-title":"Nat. Methods"},{"key":"10.1016\/j.ymthe.2024.08.016_bib52","doi-asserted-by":"crossref","first-page":"168","DOI":"10.3389\/fnmol.2020.00168","article-title":"Development and validation of CRISPR activator systems for overexpression of CB1 receptors in neurons","volume":"13","author":"Di Maria","year":"2020","journal-title":"Front. Mol. Neurosci."},{"key":"10.1016\/j.ymthe.2024.08.016_bib53","doi-asserted-by":"crossref","first-page":"28420","DOI":"10.1038\/srep28420","article-title":"Precision modulation of neurodegenerative disease-related gene expression in human iPSC-derived neurons","volume":"6","author":"Heman-Ackah","year":"2016","journal-title":"Sci. Rep."},{"key":"10.1016\/j.ymthe.2024.08.016_bib54","article-title":"Rational design of a compact CRISPR-Cas9 activator for AAV-mediated delivery","author":"Vora","year":"2018","journal-title":"bioRxiv"},{"key":"10.1016\/j.ymthe.2024.08.016_bib55","doi-asserted-by":"crossref","first-page":"1020","DOI":"10.1038\/s41593-021-00862-0","article-title":"Genome-wide CRISPRi\/a screens in human neurons link lysosomal failure to ferroptosis","volume":"24","author":"Tian","year":"2021","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.ymthe.2024.08.016_bib56","doi-asserted-by":"crossref","first-page":"5660","DOI":"10.1021\/acsnano.1c10631","article-title":"Robust, durable gene activation in\u00a0vivo via mRNA-encoded activators","volume":"16","author":"Beyersdorf","year":"2022","journal-title":"ACS Nano"},{"key":"10.1016\/j.ymthe.2024.08.016_bib57","doi-asserted-by":"crossref","first-page":"9409","DOI":"10.1073\/pnas.1707635114","article-title":"Optimized strategy for in\u00a0vivo Cas9-activation in Drosophila","volume":"114","author":"Ewen-Campen","year":"2017","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2024.08.016_bib58","doi-asserted-by":"crossref","first-page":"104954","DOI":"10.1016\/j.nbd.2020.104954","article-title":"CRISPR\/dCas9-based Scn1a gene activation in inhibitory neurons ameliorates epileptic and behavioral phenotypes of Dravet syndrome model mice","volume":"141","author":"Yamagata","year":"2020","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.ymthe.2024.08.016_bib59","doi-asserted-by":"crossref","first-page":"1716","DOI":"10.1038\/s41592-023-02036-1","article-title":"Compact engineered human mechanosensitive transactivation modules enable potent and versatile synthetic transcriptional control","volume":"20","author":"Mahata","year":"2023","journal-title":"Nat. Methods"},{"key":"10.1016\/j.ymthe.2024.08.016_bib60","doi-asserted-by":"crossref","first-page":"857","DOI":"10.1046\/j.1365-2443.2001.00469.x","article-title":"Two domains of Nrf2 cooperatively bind CBP, a CREB binding protein, and synergistically activate transcription","volume":"6","author":"Katoh","year":"2001","journal-title":"Genes Cells"},{"key":"10.1016\/j.ymthe.2024.08.016_bib61","doi-asserted-by":"crossref","first-page":"2468","DOI":"10.1038\/sj.onc.1203476","article-title":"The role of STATs in transcriptional control and their impact on cellular function","volume":"19","author":"Bromberg","year":"2000","journal-title":"Oncogene"},{"key":"10.1016\/j.ymthe.2024.08.016_bib62","doi-asserted-by":"crossref","first-page":"325","DOI":"10.1038\/s41576-021-00438-5","article-title":"Molecular mechanisms of transgenerational epigenetic inheritance","volume":"23","author":"Fitz-James","year":"2022","journal-title":"Nat. Rev. Genet."},{"key":"10.1016\/j.ymthe.2024.08.016_bib63","doi-asserted-by":"crossref","first-page":"510","DOI":"10.1038\/nbt.3199","article-title":"Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers","volume":"33","author":"Hilton","year":"2015","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib64","doi-asserted-by":"crossref","first-page":"896","DOI":"10.1038\/s41467-021-21188-2","article-title":"Programmable human histone phosphorylation and gene activation using a CRISPR\/Cas9-based chromatin kinase","volume":"12","author":"Li","year":"2021","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2024.08.016_bib65","doi-asserted-by":"crossref","first-page":"206","DOI":"10.1002\/cbic.201000195","article-title":"Structure and function of mammalian DNA methyltransferases","volume":"12","author":"Jurkowska","year":"2011","journal-title":"Chembiochem."},{"key":"10.1016\/j.ymthe.2024.08.016_bib66","doi-asserted-by":"crossref","first-page":"46545","DOI":"10.18632\/oncotarget.10234","article-title":"CRISPR-dCas9 mediated TET1 targeting for selective DNA demethylation at BRCA1 promoter","volume":"7","author":"Choudhury","year":"2016","journal-title":"Oncotarget"},{"key":"10.1016\/j.ymthe.2024.08.016_bib67","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1016\/j.cell.2016.08.056","article-title":"Editing DNA methylation in the mammalian genome","volume":"167","author":"Liu","year":"2016","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2024.08.016_bib68","doi-asserted-by":"crossref","first-page":"16009","DOI":"10.1038\/celldisc.2016.9","article-title":"A CRISPR-based approach for targeted DNA demethylation","volume":"2","author":"Xu","year":"2016","journal-title":"Cell Discov."},{"key":"10.1016\/j.ymthe.2024.08.016_bib69","doi-asserted-by":"crossref","first-page":"1060","DOI":"10.1038\/nbt.3658","article-title":"Targeted DNA demethylation in\u00a0vivo using dCas9-peptide repeat and scFv-TET1 catalytic domain fusions","volume":"34","author":"Morita","year":"2016","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib70","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1016\/j.canlet.2019.01.040","article-title":"Targeted demethylation of the SARI promotor impairs colon tumour growth","volume":"448","author":"Wang","year":"2019","journal-title":"Cancer Lett."},{"key":"10.1016\/j.ymthe.2024.08.016_bib71","doi-asserted-by":"crossref","first-page":"60535","DOI":"10.18632\/oncotarget.11142","article-title":"Waking up dormant tumor suppressor genes with zinc fingers, TALEs and the CRISPR\/dCas9 system","volume":"7","author":"Garcia-Bloj","year":"2016","journal-title":"Oncotarget"},{"key":"10.1016\/j.ymthe.2024.08.016_bib72","doi-asserted-by":"crossref","first-page":"264","DOI":"10.1089\/crispr.2021.0099","article-title":"CRISPR-mediated synergistic epigenetic and transcriptional control","volume":"5","author":"Dominguez","year":"2022","journal-title":"CRISPR J."},{"key":"10.1016\/j.ymthe.2024.08.016_bib73","doi-asserted-by":"crossref","first-page":"2503","DOI":"10.1016\/j.cell.2021.03.025","article-title":"Genome-wide programmable transcriptional memory by CRISPR-based epigenome editing","volume":"184","author":"Nu\u00f1ez","year":"2021","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2024.08.016_bib74","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1016\/j.jmb.2018.08.001","article-title":"Temporal and spatial epigenome editing allows precise gene regulation in mammalian cells","volume":"431","author":"Kuscu","year":"2019","journal-title":"J.\u00a0Mol. Biol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib75","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1038\/s41587-019-0296-7","article-title":"Dose-dependent activation of gene expression is achieved using CRISPR and small molecules that recruit endogenous chromatin machinery","volume":"38","author":"Chiarella","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib76","doi-asserted-by":"crossref","first-page":"442","DOI":"10.1016\/j.cell.2013.06.044","article-title":"CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes","volume":"154","author":"Gilbert","year":"2013","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2024.08.016_bib77","doi-asserted-by":"crossref","first-page":"1093","DOI":"10.1038\/s41592-020-0966-x","article-title":"An efficient KRAB domain for CRISPRi applications in human cells","volume":"17","author":"Alerasool","year":"2020","journal-title":"Nat. Methods"},{"key":"10.1016\/j.ymthe.2024.08.016_bib78","doi-asserted-by":"crossref","first-page":"611","DOI":"10.1038\/s41592-018-0048-5","article-title":"An enhanced CRISPR repressor for targeted mammalian gene regulation","volume":"15","author":"Yeo","year":"2018","journal-title":"Nat. Methods"},{"key":"10.1016\/j.ymthe.2024.08.016_bib79","doi-asserted-by":"crossref","first-page":"333","DOI":"10.1016\/j.cels.2017.08.011","article-title":"Multiplex enhancer interference reveals collaborative control of gene regulation by estrogen receptor \u03b1-bound enhancers","volume":"5","author":"Carleton","year":"2017","journal-title":"Cell Syst."},{"key":"10.1016\/j.ymthe.2024.08.016_bib80","doi-asserted-by":"crossref","first-page":"15315","DOI":"10.1038\/ncomms15315","article-title":"Locus-specific histone deacetylation using a synthetic CRISPR-Cas9-based HDAC","volume":"8","author":"Kwon","year":"2017","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2024.08.016_bib81","doi-asserted-by":"crossref","first-page":"221","DOI":"10.1038\/nature10805","article-title":"Enhancer decommissioning by LSD1 during embryonic stem cell differentiation","volume":"482","author":"Whyte","year":"2012","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2024.08.016_bib82","doi-asserted-by":"crossref","first-page":"1133","DOI":"10.1038\/nbt.2701","article-title":"Locus-specific editing of histone modifications at endogenous enhancers","volume":"31","author":"Mendenhall","year":"2013","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib83","doi-asserted-by":"crossref","first-page":"401","DOI":"10.1038\/nmeth.3325","article-title":"Functional annotation of native enhancers with a Cas9-histone demethylase fusion","volume":"12","author":"Kearns","year":"2015","journal-title":"Nat. Methods"},{"key":"10.1016\/j.ymthe.2024.08.016_bib84","doi-asserted-by":"crossref","first-page":"41","DOI":"10.1186\/s40035-022-00314-0","article-title":"CRISPR\/dCas9-Dnmt3a-mediated targeted DNA methylation of APP rescues brain pathology in a mouse model of Alzheimer\u2019s disease","volume":"11","author":"Park","year":"2022","journal-title":"Transl. Neurodegener."},{"key":"10.1016\/j.ymthe.2024.08.016_bib85","doi-asserted-by":"crossref","first-page":"176","DOI":"10.1186\/s13059-017-1306-z","article-title":"DNA epigenome editing using CRISPR-Cas SunTag-directed DNMT3A","volume":"18","author":"Huang","year":"2017","journal-title":"Genome Biol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib86","doi-asserted-by":"crossref","first-page":"1203","DOI":"10.1056\/NEJMoa2307798","article-title":"death after high-dose rAAV9 gene therapy in a patient with duchenne\u2019s muscular dystrophy","volume":"389","author":"Lek","year":"2023","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2024.08.016_bib87","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1038\/s41572-021-00248-3","article-title":"Duchenne muscular dystrophy","volume":"7","author":"Duan","year":"2021","journal-title":"Nat. Rev. Dis. Primers"},{"key":"10.1016\/j.ymthe.2024.08.016_bib88","doi-asserted-by":"crossref","first-page":"917","DOI":"10.1038\/s41573-023-00775-6","article-title":"Therapeutic approaches for Duchenne muscular dystrophy","volume":"22","author":"Roberts","year":"2023","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.ymthe.2024.08.016_bib89","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1111\/j.1750-3639.1996.tb00781.x","article-title":"Utrophin: a structural and functional comparison to dystrophin","volume":"6","author":"Blake","year":"1996","journal-title":"Brain Pathol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib90","doi-asserted-by":"crossref","first-page":"3333","DOI":"10.1093\/hmg\/11.26.3333","article-title":"Prevention of pathology in mdx mice by expression of utrophin: analysis using an inducible transgenic expression system","volume":"11","author":"Squire","year":"2002","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2024.08.016_bib91","doi-asserted-by":"crossref","first-page":"1441","DOI":"10.1038\/4033","article-title":"Expression of full-length utrophin prevents muscular dystrophy in mdx mice","volume":"4","author":"Tinsley","year":"1998","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2024.08.016_bib92","doi-asserted-by":"crossref","first-page":"752","DOI":"10.1093\/hmg\/ddp539","article-title":"The artificial gene Jazz, a transcriptional regulator of utrophin, corrects the dystrophic pathology in mdx mice","volume":"19","author":"di Certo","year":"2010","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2024.08.016_bib93","doi-asserted-by":"crossref","first-page":"1283","DOI":"10.1002\/jcp.24567","article-title":"Novel adeno-associated viral vector delivering the utrophin gene regulator jazz counteracts dystrophic pathology in mdx mice","volume":"229","author":"Strimpakos","year":"2014","journal-title":"J.\u00a0Cell. Physiol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib94","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1186\/1471-2199-14-3","article-title":"UtroUp is a novel six zinc finger artificial transcription factor that recognises 18 base pairs of the utrophin promoter and efficiently drives utrophin upregulation","volume":"14","author":"Onori","year":"2013","journal-title":"BMC Mol. Biol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib95","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1016\/j.ajhg.2015.11.012","article-title":"Spell checking nature: versatility of CRISPR\/Cas9 for developing treatments for inherited disorders","volume":"98","author":"Wojtal","year":"2016","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.ymthe.2024.08.016_bib96","doi-asserted-by":"crossref","first-page":"1495","DOI":"10.1016\/j.cell.2017.10.025","article-title":"In\u00a0vivo target gene activation via CRISPR\/Cas9-mediated trans-epigenetic modulation","volume":"171","author":"Liao","year":"2017","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2024.08.016_bib97","doi-asserted-by":"crossref","first-page":"4184","DOI":"10.1038\/s41467-022-31798-z","article-title":"Inactivation of Sirt6 ameliorates muscular dystrophy in mdx mice by releasing suppression of utrophin expression","volume":"13","author":"Georgieva","year":"2022","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2024.08.016_bib98","doi-asserted-by":"crossref","first-page":"7991","DOI":"10.1073\/pnas.0811599106","article-title":"Laminin-111 protein therapy prevents muscle disease in the mdx mouse model for Duchenne muscular dystrophy","volume":"106","author":"Rooney","year":"2009","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1016\/j.ymthe.2024.08.016_bib99","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1016\/j.omtn.2016.11.004","article-title":"Increased expression of laminin subunit alpha 1 chain by dCas9-VP160","volume":"6","author":"Perrin","year":"2017","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.ymthe.2024.08.016_bib100","doi-asserted-by":"crossref","first-page":"12575","DOI":"10.1038\/s41598-017-12981-5","article-title":"Timing and localization of human dystrophin isoform expression provide insights into the cognitive phenotype of Duchenne muscular dystrophy","volume":"7","author":"Doorenweerd","year":"2017","journal-title":"Sci. Rep."},{"key":"10.1016\/j.ymthe.2024.08.016_bib101","doi-asserted-by":"crossref","first-page":"232","DOI":"10.1007\/BF02265272","article-title":"Expression and regulation of the dystrophin Purkinje promoter in human skeletal muscle, heart, and brain","volume":"97","author":"Holder","year":"1996","journal-title":"Hum. Genet."},{"key":"10.1016\/j.ymthe.2024.08.016_bib102","doi-asserted-by":"crossref","first-page":"3123","DOI":"10.1016\/j.ymthe.2023.10.015","article-title":"Lethal immunotoxicity in high-dose systemic AAV therapy","volume":"31","author":"Duan","year":"2023","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2024.08.016_bib103","doi-asserted-by":"crossref","first-page":"350","DOI":"10.1089\/hum.2023.057","article-title":"Progress in bioengineering of myotropic adeno-associated viral gene therapy vectors","volume":"34","author":"Liu","year":"2023","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2024.08.016_bib104","doi-asserted-by":"crossref","first-page":"1314","DOI":"10.1002\/humu.23599","article-title":"LAMA2 gene mutation update: Toward a more comprehensive picture of the laminin-\u03b12 variome and its related phenotypes","volume":"39","author":"Oliveira","year":"2018","journal-title":"Hum. Mutat."},{"key":"10.1016\/j.ymthe.2024.08.016_bib105","doi-asserted-by":"crossref","first-page":"2690","DOI":"10.1093\/hmg\/ddl201","article-title":"Laminin \u03b11 chain improves laminin \u03b12 chain deficient peripheral neuropathy","volume":"15","author":"Gawlik","year":"2006","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2024.08.016_bib106","doi-asserted-by":"crossref","first-page":"125","DOI":"10.1038\/s41586-019-1430-x","article-title":"A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene","volume":"572","author":"Kemaladewi","year":"2019","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2024.08.016_bib107","article-title":"CRISPRa-induced upregulation of human LAMA1 compensates for LAMA2-deficiency in Merosin-deficient congenital muscular dystrophy","author":"Arockiaraj","year":"2023","journal-title":"bioRxiv"},{"key":"10.1016\/j.ymthe.2024.08.016_bib108","doi-asserted-by":"crossref","first-page":"5161","DOI":"10.1093\/nar\/gkw141","article-title":"DUX4 recruits p300\/CBP through its C-terminus and induces global H3K27 acetylation changes","volume":"44","author":"Choi","year":"2016","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.ymthe.2024.08.016_bib109","doi-asserted-by":"crossref","first-page":"527","DOI":"10.1038\/mt.2015.200","article-title":"CRISPR\/dCas9-mediated transcriptional inhibition ameliorates the epigenetic dysregulation at D4Z4 and represses DUX4-fl in FSH muscular dystrophy","volume":"24","author":"Himeda","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2024.08.016_bib110","doi-asserted-by":"crossref","first-page":"12598","DOI":"10.1038\/s41598-021-92096-0","article-title":"CRISPR mediated targeting of DUX4 distal regulatory element represses DUX4 target genes dysregulated in Facioscapulohumeral muscular dystrophy","volume":"11","author":"Das","year":"2021","journal-title":"Sci. Rep."},{"key":"10.1016\/j.ymthe.2024.08.016_bib111","doi-asserted-by":"crossref","first-page":"298","DOI":"10.1016\/j.omtm.2020.12.001","article-title":"Targeted epigenetic repression by CRISPR\/dSaCas9 suppresses pathogenic DUX4-fl expression in FSHD","volume":"20","author":"Himeda","year":"2021","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2024.08.016_bib112","doi-asserted-by":"crossref","first-page":"1797","DOI":"10.1016\/j.ymthe.2018.04.019","article-title":"Identification of epigenetic regulators of DUX4-fl for targeted therapy of facioscapulohumeral muscular dystrophy","volume":"26","author":"Himeda","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2024.08.016_bib113","doi-asserted-by":"crossref","first-page":"104533","DOI":"10.1016\/j.nbd.2019.104533","article-title":"Molecular genetics of congenital myotonic dystrophy","volume":"132","author":"Lanni","year":"2019","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.ymthe.2024.08.016_bib114","doi-asserted-by":"crossref","first-page":"857","DOI":"10.1016\/j.omtn.2023.05.007","article-title":"Specific DMPK-promoter targeting by CRISPRi reverses myotonic dystrophy type 1-associated defects in patient muscle cells","volume":"32","author":"Porquet","year":"2023","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.ymthe.2024.08.016_bib115","doi-asserted-by":"crossref","first-page":"479","DOI":"10.1016\/j.molcel.2017.09.033","article-title":"Impeding Transcription of Expanded Microsatellite Repeats by Deactivated Cas9","volume":"68","author":"Pinto","year":"2017","journal-title":"Mol. Cell"},{"key":"10.1016\/j.ymthe.2024.08.016_bib116","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":"10.1016\/j.ymthe.2024.08.016_bib117","doi-asserted-by":"crossref","first-page":"1536","DOI":"10.1038\/s41594-023-01090-9","article-title":"Elongation roadblocks mediated by dCas9 across human genes modulate transcription and nascent RNA processing","volume":"30","author":"Zukher","year":"2023","journal-title":"Nat. Struct. Mol. Biol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib118","doi-asserted-by":"crossref","first-page":"269","DOI":"10.1146\/annurev-physiol-012422-112116","article-title":"Myostatin: a skeletal muscle chalone","volume":"85","author":"Lee","year":"2023","journal-title":"Annu. Rev. Physiol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib119","doi-asserted-by":"crossref","first-page":"621","DOI":"10.1097\/WCO.0000000000000853","article-title":"The elusive promise of myostatin inhibition for muscular dystrophy","volume":"33","author":"Wagner","year":"2020","journal-title":"Curr Opin Neurol"},{"key":"10.1016\/j.ymthe.2024.08.016_bib120","doi-asserted-by":"crossref","first-page":"S153","DOI":"10.1016\/S1525-0016(16)33194-X","article-title":"385. inhibiting the myostatin signaling pathway using CRISPR\/Cas9-based repressors","volume":"24","author":"Thakore","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2024.08.016_bib121","doi-asserted-by":"crossref","first-page":"562","DOI":"10.1089\/crispr.2020.0077","article-title":"Regenerating urethral striated muscle by CRISPRi\/dCas9-KRAB-mediated myostatin silencing for obesity-associated stress urinary incontinence","volume":"3","author":"Yuan","year":"2020","journal-title":"CRISPR J."},{"key":"10.1016\/j.ymthe.2024.08.016_bib122","doi-asserted-by":"crossref","first-page":"2255","DOI":"10.1074\/jbc.271.4.2255","article-title":"Molecular cloning of caveolin-3, a novel member of the caveolin gene family expressed predominantly in muscle","volume":"271","author":"Tang","year":"1996","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.ymthe.2024.08.016_bib123","doi-asserted-by":"crossref","first-page":"3673","DOI":"10.1093\/hmg\/ddi395","article-title":"Induction of HSP70 expression and recruitment of HSC70 and HSP70 in the nucleus reduce aggregation of a polyalanine expansion mutant of PABPN1 in HeLa cells","volume":"14","author":"Wang","year":"2005","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2024.08.016_bib124","doi-asserted-by":"crossref","first-page":"12263","DOI":"10.1074\/jbc.M109633200","article-title":"Mammalian, yeast, bacterial, and chemical chaperones reduce aggregate formation and death in a cell model of oculopharyngeal muscular dystrophy","volume":"277","author":"Bao","year":"2002","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.ymthe.2024.08.016_bib125","doi-asserted-by":"crossref","first-page":"1370","DOI":"10.1038\/ng.2454","article-title":"Digenic inheritance of an SMCHD1 mutation and an FSHD-permissive D4Z4 allele causes facioscapulohumeral muscular dystrophy type 2","volume":"44","author":"Lemmers","year":"2012","journal-title":"Nat. Genet."},{"key":"10.1016\/j.ymthe.2024.08.016_bib126","doi-asserted-by":"crossref","first-page":"500","DOI":"10.1016\/j.omtn.2020.08.031","article-title":"Genome editing-mediated utrophin upregulation in duchenne muscular dystrophy stem cells","volume":"22","author":"Sengupta","year":"2020","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.ymthe.2024.08.016_bib127","doi-asserted-by":"crossref","first-page":"e29376","DOI":"10.1371\/journal.pone.0029376","article-title":"Translational regulation of utrophin by miRNAs","volume":"6","author":"Basu","year":"2011","journal-title":"PLoS One"},{"key":"10.1016\/j.ymthe.2024.08.016_bib128","doi-asserted-by":"crossref","first-page":"433","DOI":"10.1016\/bs.ctdb.2024.02.010","article-title":"Long non-coding RNAs and their role in muscle regeneration","volume":"158","author":"Biferali","year":"2024","journal-title":"Curr. Top. Dev. Biol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib129","doi-asserted-by":"crossref","first-page":"953","DOI":"10.3390\/cells9040953","article-title":"Adenoviral vectors meet gene editing: a rising partnership for the genomic engineering of human stem cells and their progeny","volume":"9","author":"Tasca","year":"2020","journal-title":"Cells"},{"key":"10.1016\/j.ymthe.2024.08.016_bib130","doi-asserted-by":"crossref","first-page":"102040","DOI":"10.1016\/j.omtn.2023.102040","article-title":"CRISPR-Cas9 delivery strategies with engineered extracellular vesicles","volume":"34","author":"Lu","year":"2023","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.ymthe.2024.08.016_bib131","doi-asserted-by":"crossref","first-page":"2129","DOI":"10.3390\/pharmaceutics14102129","article-title":"Delivery of RNAs to specific organs by lipid nanoparticles for gene therapy","volume":"14","author":"Godbout","year":"2022","journal-title":"Pharmaceutics"},{"key":"10.1016\/j.ymthe.2024.08.016_bib132","doi-asserted-by":"crossref","first-page":"746","DOI":"10.1016\/j.omtn.2023.02.025","article-title":"High-capacity adenovector delivery of forced CRISPR-Cas9 heterodimers fosters precise chromosomal deletions in human cells","volume":"31","author":"Tasca","year":"2023","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.ymthe.2024.08.016_bib133","doi-asserted-by":"crossref","first-page":"1842","DOI":"10.1038\/s41467-019-09693-x","article-title":"Multifunctional CRISPR-Cas9 with engineered immunosilenced human T\u00a0cell epitopes","volume":"10","author":"Ferdosi","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2024.08.016_bib134","doi-asserted-by":"crossref","first-page":"307","DOI":"10.2174\/156652311796150372","article-title":"Adenoviral vector immunity: its implications and circumvention strategies","volume":"11","author":"Ahi","year":"2011","journal-title":"Curr. Gene Ther."},{"key":"10.1016\/j.ymthe.2024.08.016_bib135","doi-asserted-by":"crossref","first-page":"723","DOI":"10.1016\/j.ymthe.2019.12.010","article-title":"AAV vector immunogenicity in humans: a long journey to successful gene transfer","volume":"28","author":"Verdera","year":"2020","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2024.08.016_bib136","doi-asserted-by":"crossref","first-page":"215","DOI":"10.1016\/j.jconrel.2022.09.031","article-title":"Polyethylene glycol (PEG): The nature, immunogenicity, and role in the hypersensitivity of PEGylated products","volume":"351","author":"Ibrahim","year":"2022","journal-title":"J.\u00a0Control Release"},{"key":"10.1016\/j.ymthe.2024.08.016_bib137","doi-asserted-by":"crossref","first-page":"6769","DOI":"10.1038\/s41467-021-26830-7","article-title":"Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models","volume":"12","author":"Hakim","year":"2021","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2024.08.016_bib138","doi-asserted-by":"crossref","first-page":"1191","DOI":"10.1038\/s41467-017-01222-y","article-title":"Drug-tunable multidimensional synthetic gene control using inducible degron-tagged dCas9 effectors","volume":"8","author":"Kleinjan","year":"2017","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2024.08.016_bib139","doi-asserted-by":"crossref","first-page":"3225","DOI":"10.1038\/s41467-023-38909-4","article-title":"CasTuner is a degron and CRISPR\/Cas-based toolkit for analog tuning of endogenous gene expression","volume":"14","author":"Noviello","year":"2023","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2024.08.016_bib140","doi-asserted-by":"crossref","first-page":"1043","DOI":"10.1038\/nmeth.4042","article-title":"Complex transcriptional modulation with orthogonal and inducible dCas9 regulators","volume":"13","author":"Gao","year":"2016","journal-title":"Nat. Methods"},{"key":"10.1016\/j.ymthe.2024.08.016_bib141","doi-asserted-by":"crossref","first-page":"686","DOI":"10.1021\/acssynbio.6b00313","article-title":"Orthogonal genetic regulation in human cells using chemically induced CRISPR\/Cas9 activators","volume":"6","author":"Bao","year":"2017","journal-title":"ACS Synth. Biol."},{"key":"10.1016\/j.ymthe.2024.08.016_bib142","doi-asserted-by":"crossref","first-page":"416","DOI":"10.1038\/s41586-024-07087-8","article-title":"Durable and efficient gene silencing in\u00a0vivo by hit-and-run epigenome editing","volume":"627","author":"Cappelluti","year":"2024","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2024.08.016_bib143","doi-asserted-by":"crossref","first-page":"219","DOI":"10.1016\/j.cell.2016.09.006","article-title":"Inheritable silencing of endogenous genes by hit-and-run targeted epigenetic editing","volume":"167","author":"Amabile","year":"2016","journal-title":"Cell"}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001624005392?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001624005392?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,7,2]],"date-time":"2026-07-02T19:29:13Z","timestamp":1783020553000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001624005392"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,11]]},"references-count":143,"journal-issue":{"issue":"11","published-print":{"date-parts":[[2024,11]]}},"alternative-id":["S1525001624005392"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2024.08.016","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2024,11]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"On RNA-programmable gene modulation as a versatile set of principles targeting muscular dystrophies","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2024.08.016","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2024 The Author(s). Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}]}}