{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T01:34:57Z","timestamp":1793237697003,"version":"4.5.0"},"reference-count":13,"publisher":"Elsevier BV","issue":"7","license":[{"start":{"date-parts":[[2026,7,1]],"date-time":"2026-07-01T00:00:00Z","timestamp":1782864000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,7,1]],"date-time":"2026-07-01T00:00:00Z","timestamp":1782864000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2026,7]]},"DOI":"10.1016\/j.ymthe.2026.05.022","type":"journal-article","created":{"date-parts":[[2026,6,9]],"date-time":"2026-06-09T11:53:46Z","timestamp":1781006026000},"page":"3723-3724","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"title":["\u201cJust right\u201d AAV gene therapy for spinal muscular atrophy"],"prefix":"10.1016","volume":"34","author":[{"given":"Xiupeng","family":"Chen","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9565-1567","authenticated-orcid":false,"given":"Jun","family":"Xie","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2026.05.022_bib1","first-page":"S1525","article-title":"Challenging the more-is-better dogma: A precision-optimized AAV gene therapy for SMA","volume":"15","author":"Song","year":"2026","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2026.05.022_bib2","doi-asserted-by":"crossref","first-page":"2489","DOI":"10.1016\/j.ymthe.2024.06.020","article-title":"Clinical perspectives: Treating spinal muscular atrophy","volume":"32","author":"McPheron","year":"2024","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2026.05.022_bib3","doi-asserted-by":"crossref","first-page":"15","DOI":"10.1111\/joa.12083","article-title":"Spinal muscular atrophy: a motor neuron disorder or a multi-organ disease?","volume":"224","author":"Shababi","year":"2014","journal-title":"J. Anat."},{"key":"10.1016\/j.ymthe.2026.05.022_bib4","doi-asserted-by":"crossref","first-page":"175","DOI":"10.1089\/hum.2021.116","article-title":"Biodistribution and Tolerability of AAV-PHP.B-CBh-SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles","volume":"33","author":"Palazzi","year":"2022","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2026.05.022_bib5","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1089\/hum.2018.015","article-title":"Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN","volume":"29","author":"Hinderer","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2026.05.022_bib6","doi-asserted-by":"crossref","first-page":"3005","DOI":"10.1093\/brain\/awab182","article-title":"Engineered microRNA-based regulatory element permits safe high-dose miniMECP2 gene therapy in Rett mice","volume":"144","author":"Sinnett","year":"2021","journal-title":"Brain."},{"key":"10.1016\/j.ymthe.2026.05.022_bib7","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2025.101554","article-title":"Improved AAV9-based gene therapy design for SURF1-related Leigh syndrome with minimal toxicity","volume":"33","author":"Ling","year":"2025","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2026.05.022_bib8","doi-asserted-by":"crossref","first-page":"945","DOI":"10.1038\/s44321-024-00037-x","article-title":"Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor","volume":"16","author":"Xie","year":"2024","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.ymthe.2026.05.022_bib9","doi-asserted-by":"crossref","first-page":"3547","DOI":"10.1093\/nar\/gkad1259","article-title":"A super minigene with a short promoter and truncated introns recapitulates essential features of transcription and splicing regulation of the SMN1 and SMN2 genes","volume":"52","author":"Ottesen","year":"2024","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.ymthe.2026.05.022_bib10","doi-asserted-by":"crossref","first-page":"228","DOI":"10.1016\/j.bbrc.2010.09.131","article-title":"Targeted methylation of CMV and E1A viral promoters","volume":"402","author":"Hsu","year":"2010","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"10.1016\/j.ymthe.2026.05.022_bib11","doi-asserted-by":"crossref","first-page":"1363","DOI":"10.1016\/j.ymthe.2017.03.028","article-title":"Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity","volume":"25","author":"Xie","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2026.05.022_bib12","doi-asserted-by":"crossref","first-page":"870","DOI":"10.1172\/JCI79213","article-title":"Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy","volume":"125","author":"Chandler","year":"2015","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.ymthe.2026.05.022_bib13","doi-asserted-by":"crossref","first-page":"1756","DOI":"10.1016\/j.ymthe.2020.07.006","article-title":"Quantification of CpG Motifs in rAAV Genomes: Avoiding the Toll","volume":"28","author":"Wright","year":"2020","journal-title":"Mol. Ther."}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001626004090?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001626004090?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T01:27:13Z","timestamp":1793237233000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001626004090"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,7]]},"references-count":13,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2026,7]]}},"alternative-id":["S1525001626004090"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2026.05.022","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2026,7]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"\u201cJust right\u201d AAV gene therapy for spinal muscular atrophy","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2026.05.022","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2026.04.027","name":"associatedlink","label":"CrossRef DOI link to the associated document"},{"value":"simple-article","name":"content_type","label":"Content Type"},{"value":"Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}]}}