{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,3,27]],"date-time":"2025-03-27T04:27:10Z","timestamp":1743049630677,"version":"3.40.3"},"publisher-location":"New York, NY","reference-count":59,"publisher":"Springer US","isbn-type":[{"type":"print","value":"9781071617519"},{"type":"electronic","value":"9781071617526"}],"license":[{"start":{"date-parts":[[2021,11,12]],"date-time":"2021-11-12T00:00:00Z","timestamp":1636675200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"},{"start":{"date-parts":[[2021,11,12]],"date-time":"2021-11-12T00:00:00Z","timestamp":1636675200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2022]]},"DOI":"10.1007\/978-1-0716-1752-6_31","type":"book-chapter","created":{"date-parts":[[2021,11,11]],"date-time":"2021-11-11T21:02:28Z","timestamp":1636664548000},"page":"491-513","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Evaluating Efficacy of Peptide-Delivered Oligonucleotides Using the Severe Taiwanese SMA Mouse Model"],"prefix":"10.1007","author":[{"given":"Larissa","family":"Goli","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jessica","family":"Stoodley","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Suzan M.","family":"Hammond","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Richard","family":"Raz","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2021,11,12]]},"reference":[{"key":"31_CR1","doi-asserted-by":"publisher","first-page":"9","DOI":"10.1038\/nrneurol.2017.148","volume":"14","author":"C Rinaldi","year":"2018","unstructured":"Rinaldi C, Wood MJA (2018) Antisense oligonucleotides: the next frontier for treatment of neurological disorders. Nat Rev Neurol 14:9\u201321. https:\/\/doi.org\/10.1038\/nrneurol.2017.148","journal-title":"Nat Rev Neurol"},{"key":"31_CR2","doi-asserted-by":"publisher","first-page":"605","DOI":"10.1146\/annurev-pharmtox-010818-021050","volume":"59","author":"CIE Smith","year":"2019","unstructured":"Smith CIE, Zain R (2019) Therapeutic oligonucleotides: state of the art. Annu Rev Pharmacol Toxicol 59:605\u2013630. https:\/\/doi.org\/10.1146\/annurev-pharmtox-010818-021050","journal-title":"Annu Rev Pharmacol Toxicol"},{"issue":"10","key":"31_CR3","doi-asserted-by":"publisher","first-page":"673","DOI":"10.1038\/s41573-020-0075-7","volume":"19","author":"TC Roberts","year":"2020","unstructured":"Roberts TC, Langer R, Wood MJA (2020) Advances in oligonucleotide drug delivery. Nat Rev Drug Discov 19(10):673\u2013694. https:\/\/doi.org\/10.1038\/s41573-020-0075-7","journal-title":"Nat Rev Drug Discov"},{"key":"31_CR4","doi-asserted-by":"publisher","first-page":"284","DOI":"10.1016\/j.bcp.2009.04.013","volume":"78","author":"RS Geary","year":"2009","unstructured":"Geary RS, Wancewicz E, Matson J, Pearce M, Siwkowski A, Swayze E, Bennett F (2009) Effect of dose and plasma concentration on liver uptake and pharmacologic activity of a 2\u2032-methoxyethyl modified chimeric antisense oligonucleotide targeting PTEN. Biochem Pharmacol 78:284\u2013291. https:\/\/doi.org\/10.1016\/j.bcp.2009.04.013","journal-title":"Biochem Pharmacol"},{"key":"31_CR5","doi-asserted-by":"publisher","first-page":"1308","DOI":"10.1021\/js9800836","volume":"87","author":"RJ Boado","year":"1998","unstructured":"Boado RJ, Tsukamoto H, Pardridge WM (1998) Drug delivery of antisense molecules to the brain for treatment of Alzheimer\u2019s disease and cerebral AIDS. J Pharm Sci 87:1308\u20131315","journal-title":"J Pharm Sci"},{"key":"31_CR6","doi-asserted-by":"publisher","first-page":"1584","DOI":"10.1038\/nm.3407","volume":"19","author":"B Obermeier","year":"2013","unstructured":"Obermeier B, Daneman R, Ransohoff RM (2013) Development, maintenance and disruption of the blood-brain barrier. Nat Med 19:1584\u20131596. https:\/\/doi.org\/10.1038\/nm.3407","journal-title":"Nat Med"},{"key":"31_CR7","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1016\/j.neuron.2017.07.030","volume":"96","author":"C Iadecola","year":"2017","unstructured":"Iadecola C (2017) The neurovascular unit coming of age: a journey through neurovascular coupling in health and disease. Neuron 96:17\u201342. https:\/\/doi.org\/10.1016\/j.neuron.2017.07.030","journal-title":"Neuron"},{"key":"31_CR8","doi-asserted-by":"publisher","first-page":"899","DOI":"10.1177\/0883073815627882","volume":"31","author":"M Hache","year":"2016","unstructured":"Hache M, Swoboda KJ, Sethna N, Farrow-Gillespie A, Khandji A, Xia S, Bishop KM (2016) Intrathecal injections in children with spinal muscular atrophy: nusinersen clinical trial experience. J Child Neurol 31:899\u2013906. https:\/\/doi.org\/10.1177\/0883073815627882","journal-title":"J Child Neurol"},{"key":"31_CR9","doi-asserted-by":"publisher","first-page":"3017","DOI":"10.1016\/s0140-6736(16)31408-8","volume":"388","author":"RS Finkel","year":"2016","unstructured":"Finkel RS, Chiriboga CA, Vajsar J, Day JW, Montes J, De Vivo DC, Yamashita M, Rigo F, Hung G, Schneider E, Norris DA, Xia S, Bennett CF, Bishop KM (2016) Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet 388:3017\u20133026. https:\/\/doi.org\/10.1016\/s0140-6736(16)31408-8","journal-title":"Lancet"},{"key":"31_CR10","doi-asserted-by":"publisher","first-page":"1189","DOI":"10.1016\/0092-8674(88)90263-2","volume":"55","author":"AD Frankel","year":"1988","unstructured":"Frankel AD, Pabo CO (1988) Cellular uptake of the tat protein form human immunodeficiency virus. Cell 55:1189\u20131193","journal-title":"Cell"},{"key":"31_CR11","doi-asserted-by":"publisher","first-page":"18188","DOI":"10.1074\/jbc.271.30.18188","volume":"271","author":"D Derossi","year":"1996","unstructured":"Derossi D, Calvet S, Trembleau A, Brunissen A, Chassaing G, Prochiantz A (1996) Cell internalization of the third helix of the Antennapedia homeodomain is receptor-independent. J Biol Chem 271:18188\u201318193","journal-title":"J Biol Chem"},{"key":"31_CR12","doi-asserted-by":"publisher","first-page":"3612","DOI":"10.1021\/jm0105676","volume":"45","author":"JB Rothbard","year":"2002","unstructured":"Rothbard JB, Kreider E, VanDeusen CL, Wright L, Wylie BL, Wender PA (2002) Arginine-rich molecular transporters for drug delivery: role of backbone spacing in cellular uptake. J Med Chem 45:3612\u20133618. https:\/\/doi.org\/10.1021\/jm0105676","journal-title":"J Med Chem"},{"key":"31_CR13","doi-asserted-by":"publisher","first-page":"189","DOI":"10.1016\/j.jconrel.2005.09.012","volume":"110","author":"S El-Andaloussi","year":"2005","unstructured":"El-Andaloussi S, Johansson H, Magnusdottir A, J\u00e4rver P, Lundberg P, Langel \u00dc (2005) TP10, a delivery vector for decoy oligonucleotides targeting the Myc protein. J Control Release 110:189\u2013201. https:\/\/doi.org\/10.1016\/j.jconrel.2005.09.012","journal-title":"J Control Release"},{"key":"31_CR14","doi-asserted-by":"publisher","first-page":"455","DOI":"10.1002\/psc","volume":"14","author":"R Abes","year":"2008","unstructured":"Abes R, Arzumanov A, Moulton H, Abes S, Ivanova G, Gait MJ, Iversen P, Lebleu B (2008) Arginine-rich cell penetrating peptides: design, structure\u2013activity, and applications to alter pre-mRNA splicing by steric-block oligonucleotides. J Pept Sci 14:455\u2013460. https:\/\/doi.org\/10.1002\/psc","journal-title":"J Pept Sci"},{"key":"31_CR15","doi-asserted-by":"publisher","first-page":"6418","DOI":"10.1093\/nar\/gkn671","volume":"36","author":"GD Ivanova","year":"2008","unstructured":"Ivanova GD, Arzumanov A, Abes R, Yin H, Wood MJA, Lebleu B, Gait MJ (2008) Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle. Nucleic Acids Res 36:6418\u20136428. https:\/\/doi.org\/10.1093\/nar\/gkn671","journal-title":"Nucleic Acids Res"},{"key":"31_CR16","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0045501","volume":"7","author":"C Heffernan","year":"2012","unstructured":"Heffernan C, Sumer H, Guillemin GJ, Manuelpillai U, Verma PJ (2012) Design and screening of a glial cell-specific, cell penetrating peptide for therapeutic applications in multiple sclerosis. PLoS One 7:e45501. https:\/\/doi.org\/10.1371\/journal.pone.0045501","journal-title":"PLoS One"},{"key":"31_CR17","doi-asserted-by":"publisher","first-page":"1810","DOI":"10.1074\/jbc.M117.815373","volume":"293","author":"JL Larabee","year":"2018","unstructured":"Larabee JL, Hauck GD, Ballard JD (2018) Cell-penetrating peptides derived from Clostridium difficile TcdB2 and a related large clostridial toxin. J Biol Chem 293:1810\u20131819. https:\/\/doi.org\/10.1074\/jbc.M117.815373","journal-title":"J Biol Chem"},{"key":"31_CR18","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1038\/srep11719","volume":"5","author":"HY Kim","year":"2015","unstructured":"Kim HY, Yum SY, Jang G, Ahn DR (2015) Discovery of a non-cationic cell penetrating peptide derived from membrane-interacting human proteins and its potential as a protein delivery carrier. Sci Rep 5:1\u201315. https:\/\/doi.org\/10.1038\/srep11719","journal-title":"Sci Rep"},{"key":"31_CR19","doi-asserted-by":"publisher","first-page":"1","DOI":"10.3390\/molecules24050927","volume":"24","author":"J Habault","year":"2019","unstructured":"Habault J, Poyet J-L (2019) Recent advances in cell penetrating peptide-based anticancer therapies. Molecules 24:1\u201317. https:\/\/doi.org\/10.3390\/molecules24050927","journal-title":"Molecules"},{"key":"31_CR20","doi-asserted-by":"publisher","first-page":"556","DOI":"10.1021\/bc900445g","volume":"22","author":"MP Gillmeister","year":"2011","unstructured":"Gillmeister MP, Betenbaugh MJ, Fishman PS (2011) Cellular trafficking and photochemical internalization of cell penetrating peptide linked cargo proteins: a dual fluorescent labeling study. Bioconjug Chem 22:556\u2013566. https:\/\/doi.org\/10.1021\/bc900445g","journal-title":"Bioconjug Chem"},{"key":"31_CR21","doi-asserted-by":"publisher","first-page":"1","DOI":"10.3390\/ijms17020185","volume":"17","author":"M Kristensen","year":"2016","unstructured":"Kristensen M, Birch D, Morck Nielsen H (2016) Applications and challenges for use of cell-penetrating peptides as delivery vectors for peptide and protein cargos. Int J Mol Sci 17:1\u201317. https:\/\/doi.org\/10.3390\/ijms17020185","journal-title":"Int J Mol Sci"},{"issue":"Pt 4","key":"31_CR22","doi-asserted-by":"publisher","first-page":"775","DOI":"10.1042\/BST0350775","volume":"35","author":"R Abes","year":"2007","unstructured":"Abes R, Arzumanov AA, Moulton HM, Abes S, Ivanova GD, Iversen PL, Gait MJ, Lebleu B (2007) Cell-penetrating-peptide-based delivery of oligonucleotides: an overview. Biochem Soc Trans 35(Pt 4):775\u2013779","journal-title":"Biochem Soc Trans"},{"key":"31_CR23","doi-asserted-by":"publisher","first-page":"1","DOI":"10.3390\/biomedicines6020051","volume":"6","author":"G McClorey","year":"2018","unstructured":"McClorey G, Banerjee S (2018) Cell-penetrating peptides to enhance delivery of oligonucleotide-based therapeutics. Biomedicine 6:1\u201315. https:\/\/doi.org\/10.3390\/biomedicines6020051","journal-title":"Biomedicine"},{"key":"31_CR24","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1089\/nat.2018.0747","volume":"29","author":"MJ Gait","year":"2019","unstructured":"Gait MJ, Arzumanov AA, McClorey G, Godfrey C, Betts C, Hammond S, Wood MJA (2019) Cell-penetrating peptide conjugates of steric blocking oligonucleotides as therapeutics for neuromuscular diseases from a historical perspective to current prospects of treatment. Nucleic Acid Ther 29:1\u201312. https:\/\/doi.org\/10.1089\/nat.2018.0747","journal-title":"Nucleic Acid Ther"},{"key":"31_CR25","doi-asserted-by":"publisher","first-page":"520","DOI":"10.1038\/gt.2017.34","volume":"24","author":"NN Singh","year":"2017","unstructured":"Singh NN, Howell MD, Androphy EJ, Singh RN (2017) How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy. Gene Ther 24:520\u2013526. https:\/\/doi.org\/10.1038\/gt.2017.34","journal-title":"Gene Ther"},{"key":"31_CR26","doi-asserted-by":"publisher","first-page":"223","DOI":"10.1007\/s00401-008-0411-1","volume":"116","author":"G Simic","year":"2008","unstructured":"Simic G (2008) Pathogenesis of proximal autosomal recessive spinal muscular atrophy. Acta Neuropathol 116:223\u2013234. https:\/\/doi.org\/10.1007\/s00401-008-0411-1","journal-title":"Acta Neuropathol"},{"key":"31_CR27","doi-asserted-by":"publisher","first-page":"979","DOI":"10.1001\/archneurol.2011.74","volume":"68","author":"SJ Kolb","year":"2011","unstructured":"Kolb SJ, Kissel JT (2011) Spinal muscular atrophy: a timely review. Arch Neurol 68:979\u2013984. https:\/\/doi.org\/10.1001\/archneurol.2011.74","journal-title":"Arch Neurol"},{"key":"31_CR28","doi-asserted-by":"publisher","first-page":"155","DOI":"10.1016\/0092-8674(95)90460-3","volume":"80","author":"S Lefebvre","year":"1995","unstructured":"Lefebvre S, B\u00fcrglen L, Reboullet S, Clermont O, Burlet P, Viollet L, Benichou B, Cruaud C, Millasseau P, Zeviani M, Le Paslier D, Fr\u00e9zal J, Cohen D, Weissenbach J, Munnich A, Melki J (1995) Identification and characterization of a spinal muscular atrophy-determining gene. Cell 80:155\u2013165","journal-title":"Cell"},{"unstructured":"FDA (2016) FDA approves first drug for spinal muscular atrophy \u2014 New therapy addresses unmet medical need for rare disease. In: [Press release]. https:\/\/www.fda.gov\/newsevents\/newsroom\/pressannouncements\/ucm534611.htm. Accessed 4 Sep 2018","key":"31_CR29"},{"unstructured":"EMA (2017) First medicine for spinal muscular atrophy. https:\/\/www.ema.europa.eu\/en\/news\/first-medicine-spinal-muscular-atrophy. Accessed 4 Sep 2018","key":"31_CR30"},{"unstructured":"PMDA (2017) Report on the Deliberation Results on the Approval of Nusinersen. https:\/\/www.pmda.go.jp\/files\/000228334.pdf. Accessed 17 Dec 2020","key":"31_CR31"},{"key":"31_CR32","doi-asserted-by":"publisher","first-page":"1153","DOI":"10.2147\/TCRM.S172291","volume":"15","author":"T Dangouloff","year":"2019","unstructured":"Dangouloff T, Servais L (2019) Clinical evidence supporting early treatment of patients with spinal muscular atrophy: current perspectives. Ther Clin Risk Manag 15:1153\u20131161. https:\/\/doi.org\/10.2147\/TCRM.S172291","journal-title":"Ther Clin Risk Manag"},{"unstructured":"NOVARTIS (2020) AveXis receives positive CHMP opinion for Zolgensma\u00ae, the only gene therapy for spinal muscular atrophy (SMA). https:\/\/www.novartis.com\/news\/media-releases\/avexis-receives-positive-chmp-opinion-zolgensma-only-gene-therapy-spinal-muscular-atrophy-sma. Accessed 7 Dec 2020","key":"31_CR33"},{"unstructured":"Genentech (2020) FDA approves Genentech\u2019s Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older. https:\/\/www.gene.com\/media\/press-releases\/14866\/2020-08-07\/fda-approves-genentechs-evrysdi-risdipla. Accessed 12 July 2020","key":"31_CR34"},{"key":"31_CR35","doi-asserted-by":"publisher","first-page":"66","DOI":"10.1038\/71709","volume":"24","author":"HM Hsieh-Li","year":"2000","unstructured":"Hsieh-Li HM, Chang JG, Jong YJ, Wu MH, Wang NM, Tsai CH, Li H, Hsieh-Li HM, Chang JG, Jong YJ, Wu MH, Wang NM, Tsai CH, Li H (2000) A mouse model for spinal muscular atrophy. Nat Genet 24:66\u201370","journal-title":"Nat Genet"},{"key":"31_CR36","doi-asserted-by":"publisher","first-page":"517","DOI":"10.1016\/j.bbrc.2009.11.090","volume":"391","author":"RG Gogliotti","year":"2010","unstructured":"Gogliotti RG, Hammond SM, Lutz C, Didonato CJ (2010) Molecular and phenotypic reassessment of an infrequently used mouse model for spinal muscular atrophy. Biochem Biophys Res Commun 391:517\u2013522. https:\/\/doi.org\/10.1016\/j.bbrc.2009.11.090","journal-title":"Biochem Biophys Res Commun"},{"key":"31_CR37","doi-asserted-by":"publisher","first-page":"457","DOI":"10.1242\/dmm.007245","volume":"4","author":"JN Sleigh","year":"2011","unstructured":"Sleigh JN, Gillingwater TH, Talbot K (2011) The contribution of mouse models to understanding the pathogenesis of spinal muscular atrophy. Dis Model Mech 4:457\u2013467. https:\/\/doi.org\/10.1242\/dmm.007245","journal-title":"Dis Model Mech"},{"key":"31_CR38","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1371\/journal.pone.0154723","volume":"11","author":"TL Lin","year":"2016","unstructured":"Lin TL, Chen TH, Hsu YY, Cheng YH, Juang BT, Jong YJ (2016) Selective neuromuscular denervation in Taiwanese severe SMA mouse can be reversed by morpholino antisense oligonucleotides. PLoS One 11:1\u201315. https:\/\/doi.org\/10.1371\/journal.pone.0154723","journal-title":"PLoS One"},{"key":"31_CR39","doi-asserted-by":"publisher","first-page":"643","DOI":"10.1038\/ejhg.2012.222","volume":"21","author":"J Schreml","year":"2013","unstructured":"Schreml J, Riessland M, Paterno M, Garbes L, Robach K, Ackermann B, Kr\u00e4mer J, Somers E, Parson SH, Heller R, Berkessel A, Sterner-Kock A, Wirth B (2013) Severe SMA mice show organ impairment that cannot be rescued by therapy with the HDACi JNJ-26481585. Eur J Hum Genet 21:643\u2013652. https:\/\/doi.org\/10.1038\/ejhg.2012.222","journal-title":"Eur J Hum Genet"},{"key":"31_CR40","doi-asserted-by":"publisher","first-page":"845","DOI":"10.1093\/hmg\/ddi078","volume":"14","author":"TT Le","year":"2005","unstructured":"Le TT, Pham LT, Butchbach MER, Zhang HL, Monani UR, Coovert DD, Gavrilina TO, Xing L, Bassell GJ, Burghes AHM (2005) SMN\u03947, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN. Hum Mol Genet 14:845\u2013857. https:\/\/doi.org\/10.1093\/hmg\/ddi078","journal-title":"Hum Mol Genet"},{"key":"31_CR41","doi-asserted-by":"publisher","first-page":"1634","DOI":"10.1101\/gad.1941310.assembly","volume":"24","author":"Y Hua","year":"2010","unstructured":"Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF, Krainer AR (2010) Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes Dev 24:1634\u20131644. https:\/\/doi.org\/10.1101\/gad.1941310.assembly","journal-title":"Genes Dev"},{"key":"31_CR42","doi-asserted-by":"publisher","first-page":"123","DOI":"10.1038\/nature10485","volume":"478","author":"Y Hua","year":"2011","unstructured":"Hua Y, Sahashi K, Rigo F, Hung G, Horev G, Bennett CF, Krainer AR (2011) Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model. Nature 478:123\u2013126. https:\/\/doi.org\/10.1038\/nature10485","journal-title":"Nature"},{"key":"31_CR43","doi-asserted-by":"publisher","first-page":"288","DOI":"10.1101\/gad.256644.114","volume":"29","author":"Y Hua","year":"2015","unstructured":"Hua Y, Liu YH, Sahashi K, Rigo F, Bennett CF, Krainer AR (2015) Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models. Genes Dev 29:288\u2013297. https:\/\/doi.org\/10.1101\/gad.256644.114","journal-title":"Genes Dev"},{"key":"31_CR44","doi-asserted-by":"publisher","first-page":"10962","DOI":"10.1073\/pnas.1605731113","volume":"113","author":"SM Hammond","year":"2016","unstructured":"Hammond SM, Hazell G, Shabanpoor F, Saleh AF, Bowerman M, Sleigh JN, Meijboom KE, Zhou H, Muntoni F, Talbot K, Gait MJ, Wood MJ (2016) Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy. Proc Natl Acad Sci U S A 113:10962\u201310967. https:\/\/doi.org\/10.1073\/pnas.1605731113","journal-title":"Proc Natl Acad Sci U S A"},{"key":"31_CR45","doi-asserted-by":"publisher","first-page":"221","DOI":"10.1007\/978-1-4939-9670-4_13","volume":"2036","author":"SM Hammond","year":"2019","unstructured":"Hammond SM, Abendroth F, Gait MJ, Wood MJA (2019) Evaluation of cell-penetrating peptide delivery of antisense oligonucleotides for therapeutic efficacy in spinal muscular atrophy. Methods Mol Biol 2036:221\u2013236. https:\/\/doi.org\/10.1007\/978-1-4939-9670-4_13","journal-title":"Methods Mol Biol"},{"key":"31_CR46","doi-asserted-by":"publisher","first-page":"66","DOI":"10.1016\/j.neuron.2016.11.033","volume":"93","author":"C D\u2019Ydewalle","year":"2017","unstructured":"D\u2019Ydewalle C, Ramos DM, Pyles NJ, Ng SY, Gorz M, Pilato CM, Ling K, Kong L, Ward AJ, Rubin LL, Rigo F, Bennett CF, Sumner CJ (2017) The antisense transcript SMN-AS1 regulates SMN expression and is a novel therapeutic target for spinal muscular atrophy. Neuron 93:66\u201379. https:\/\/doi.org\/10.1016\/j.neuron.2016.11.033","journal-title":"Neuron"},{"key":"31_CR47","doi-asserted-by":"publisher","first-page":"4","DOI":"10.1002\/psc.2836","volume":"22","author":"R Behrendt","year":"2016","unstructured":"Behrendt R, White P, Offer J (2016) Advances in Fmoc solid-phase peptide synthesis. J Pept Sci 22:4\u201327. https:\/\/doi.org\/10.1002\/psc.2836","journal-title":"J Pept Sci"},{"key":"31_CR48","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1007\/978-1-0716-0928-6_1","volume":"2208","author":"R Raz","year":"2021","unstructured":"Raz R, Offer J (2021) A shortcut to the synthesis of peptide thioesters. Methods Mol Biol 2208:1\u201312. https:\/\/doi.org\/10.1007\/978-1-0716-0928-6_1","journal-title":"Methods Mol Biol"},{"key":"31_CR49","doi-asserted-by":"publisher","first-page":"13","DOI":"10.1007\/978-1-61779-111-6_2","volume":"57","author":"S Spijker","year":"2011","unstructured":"Spijker S (2011) Dissection of rodent brain regions. NeuroMethods 57:13\u201326. https:\/\/doi.org\/10.1007\/978-1-61779-111-6_2","journal-title":"NeuroMethods"},{"key":"31_CR50","doi-asserted-by":"publisher","first-page":"275","DOI":"10.1089\/nat.2014.0528","volume":"25","author":"U Burki","year":"2015","unstructured":"Burki U, Keane J, Blain A, O\u2019Donovan L, Gait MJ, Laval SH, Straub V (2015) Development and application of an ultrasensitive hybridization-based ELISA method for the determination of peptide-conjugated phosphorodiamidate morpholino oligonucleotides. Nucleic Acid Ther 25:275\u2013284. https:\/\/doi.org\/10.1089\/nat.2014.0528","journal-title":"Nucleic Acid Ther"},{"key":"31_CR51","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/29.9.e45","volume":"29","author":"MW Pfaffl","year":"2001","unstructured":"Pfaffl MW (2001) A new mathematical model for relative quantification in real-time RT-PCR. Nucleic Acids Res 29:e45","journal-title":"Nucleic Acids Res"},{"key":"31_CR52","doi-asserted-by":"publisher","first-page":"135","DOI":"10.3233\/JND-180315","volume":"5","author":"A Pechmann","year":"2018","unstructured":"Pechmann A, Langer T, Schorling D, Stein S, Vogt S, Schara U, K\u00f6lbel H, Schwartz O, Hahn A, Giese K, Johannsen J, Denecke J, Wei\u00df C, Theophil M, Kirschner J (2018) Evaluation of children with SMA type 1 under treatment with nusinersen within the expanded access program in Germany. J Neuromuscul Dis 5:135\u2013143. https:\/\/doi.org\/10.3233\/JND-180315","journal-title":"J Neuromuscul Dis"},{"key":"31_CR53","doi-asserted-by":"publisher","first-page":"1325","DOI":"10.1021\/bc070060v","volume":"18","author":"A Amantana","year":"2007","unstructured":"Amantana A, Moulton HM, Cate ML, Reddy MT, Whitehead T, Hassinger JN, Youngblood DS, Iversen PL (2007) Pharmacokinetics, biodistribution, stability and toxicity of a cell-penetrating peptide-morpholino oligomer conjugate. Bioconjug Chem 18:1325\u20131331","journal-title":"Bioconjug Chem"},{"key":"31_CR54","doi-asserted-by":"publisher","first-page":"4225","DOI":"10.1093\/hmg\/ddv155","volume":"24","author":"C Godfrey","year":"2015","unstructured":"Godfrey C, Muses S, McClorey G, Wells KE, Coursindel T, Terry RL, Betts C, Hammond S, O\u2019Donovan L, Hildyard J, El Andaloussi S, Gait MJ, Wood MJ, Wells DJ (2015) How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse. Hum Mol Genet 24:4225\u20134237. https:\/\/doi.org\/10.1093\/hmg\/ddv155","journal-title":"Hum Mol Genet"},{"key":"31_CR55","first-page":"633","volume-title":"Clinical pharmacokinetics and pharmacodynamics","author":"M Rowland","year":"2011","unstructured":"Rowland M, Tozer TN (2011) Chapter 21: Protein drugs. In: Clinical pharmacokinetics and pharmacodynamics. Lippincott Wiliams & Wilkins, London, pp 633\u2013662"},{"key":"31_CR56","doi-asserted-by":"publisher","first-page":"143","DOI":"10.1007\/978-1-59745-060-7_9","volume":"482","author":"V Shinin","year":"2009","unstructured":"Shinin V, Gayraud-Morel B, Tajbakhsh S (2009) Template DNA-strand co-segregation and asymetric cell division in skeletal muscle stem cells. Methods Mol Biol 482:143\u2013158","journal-title":"Methods Mol Biol"},{"key":"31_CR57","doi-asserted-by":"publisher","first-page":"104","DOI":"10.1016\/j.mrgentox.2011.12.017","volume":"15","author":"JC Corton","year":"2012","unstructured":"Corton JC, Bushel PR, Fostel J, O\u2019Lone RB (2012) Sources of variance in baseline gene expression in the rodent liver. Mutat Res 15:104\u2013112. https:\/\/doi.org\/10.1016\/j.mrgentox.2011.12.017","journal-title":"Mutat Res"},{"key":"31_CR58","doi-asserted-by":"publisher","first-page":"4","DOI":"10.1038\/s41598-018-26431-3","volume":"8","author":"PEL Day","year":"2018","unstructured":"Day PEL, Chambers KF, Winterbone MS, Garc\u00eda-Blanco T, Vauzour D, Kroon PA (2018) Validation of control genes and a standardised protocol for quantifying gene expression in the livers of C57BL\/6 and ApoE\u2212\/\u2212mice. Sci Rep 8:4\u201311. https:\/\/doi.org\/10.1038\/s41598-018-26431-3","journal-title":"Sci Rep"},{"key":"31_CR59","doi-asserted-by":"publisher","first-page":"132","DOI":"10.22074\/cellj.2018.5266","volume":"20","author":"WL Zhang","year":"2018","unstructured":"Zhang WL, Liu SH, Zhang WC, Hu W, Jiang M, Tamadon A, Feng Y (2018) Skeletal muscle CLARITY: a preliminary study of imaging the three-dimensional architecture of blood vessels and neurons. Cell J 20:132\u2013137. https:\/\/doi.org\/10.22074\/cellj.2018.5266","journal-title":"Cell J"}],"container-title":["Methods in Molecular Biology","Cell Penetrating Peptides"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/978-1-0716-1752-6_31","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,11,11]],"date-time":"2021-11-11T21:10:59Z","timestamp":1636665059000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/978-1-0716-1752-6_31"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,11,12]]},"ISBN":["9781071617519","9781071617526"],"references-count":59,"URL":"https:\/\/doi.org\/10.1007\/978-1-0716-1752-6_31","relation":{},"ISSN":["1064-3745","1940-6029"],"issn-type":[{"type":"print","value":"1064-3745"},{"type":"electronic","value":"1940-6029"}],"subject":[],"published":{"date-parts":[[2021,11,12]]},"assertion":[{"value":"12 November 2021","order":1,"name":"first_online","label":"First Online","group":{"name":"ChapterHistory","label":"Chapter History"}}]}}