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Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.
Birch SM, Lawlor MW, Conlon TJ, Guo LJ, Crudele JM, Hawkins EC, Nghiem PP, Ahn M, Meng H, Beatka MJ, Fickau BA, Prieto JC, Styner MA, Struharik MJ, Shanks C, Brown KJ, Golebiowski D, Bettis AK, Balog-Alvarez CJ, Clement N, Coleman KE, Corti M, Pan X, Hauschka SD, Gonzalez JP, Morris CA, Schneider JS, Duan D, Chamberlain JS, Byrne BJ, Kornegay JN. Birch SM, et al. Among authors: pan x. Sci Transl Med. 2023 Jan 4;15(677):eabo1815. doi: 10.1126/scitranslmed.abo1815. Epub 2023 Jan 4. Sci Transl Med. 2023. PMID: 36599002
Dystrophin contains multiple independent membrane-binding domains.
Zhao J, Kodippili K, Yue Y, Hakim CH, Wasala L, Pan X, Zhang K, Yang NN, Duan D, Lai Y. Zhao J, et al. Among authors: pan x. Hum Mol Genet. 2016 Sep 1;25(17):3647-3653. doi: 10.1093/hmg/ddw210. Epub 2016 Jul 4. Hum Mol Genet. 2016. PMID: 27378693 Free PMC article.
A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy.
Hakim CH, Wasala NB, Pan X, Kodippili K, Yue Y, Zhang K, Yao G, Haffner B, Duan SX, Ramos J, Schneider JS, Yang NN, Chamberlain JS, Duan D. Hakim CH, et al. Among authors: pan x. Mol Ther Methods Clin Dev. 2017 Jul 27;6:216-230. doi: 10.1016/j.omtm.2017.06.006. eCollection 2017 Sep 15. Mol Ther Methods Clin Dev. 2017. PMID: 28932757 Free PMC article.
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