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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: chamberlain js. Sci Transl Med. 2023 Jan 4;15(677):eabo1815. doi: 10.1126/scitranslmed.abo1815. Epub 2023 Jan 4. Sci Transl Med. 2023. PMID: 36599002 Free PMC article.
Viral vectors for gene transfer of micro-, mini-, or full-length dystrophin.
Scott JM, Li S, Harper SQ, Welikson R, Bourque D, DelloRusso C, Hauschka SD, Chamberlain JS. Scott JM, et al. Among authors: chamberlain js. Neuromuscul Disord. 2002 Oct;12 Suppl 1:S23-9. doi: 10.1016/s0960-8966(02)00078-0. Neuromuscul Disord. 2002. PMID: 12206791 Review.
Gene therapy of muscular dystrophy.
Chamberlain JS. Chamberlain JS. Hum Mol Genet. 2002 Oct 1;11(20):2355-62. doi: 10.1093/hmg/11.20.2355. Hum Mol Genet. 2002. PMID: 12351570 Review.
249 results