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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: byrne bj. 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.
Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults.
Brantly ML, Spencer LT, Humphries M, Conlon TJ, Spencer CT, Poirier A, Garlington W, Baker D, Song S, Berns KI, Muzyczka N, Snyder RO, Byrne BJ, Flotte TR. Brantly ML, et al. Among authors: byrne bj. Hum Gene Ther. 2006 Dec;17(12):1177-86. doi: 10.1089/hum.2006.17.1177. Hum Gene Ther. 2006. PMID: 17115945 Clinical Trial.
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.
Hauswirth WW, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, Wang L, Conlon TJ, Boye SL, Flotte TR, Byrne BJ, Jacobson SG. Hauswirth WW, et al. Among authors: byrne bj. Hum Gene Ther. 2008 Oct;19(10):979-90. doi: 10.1089/hum.2008.107. Hum Gene Ther. 2008. PMID: 18774912 Free PMC article. Clinical Trial.
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy.
Brantly ML, Chulay JD, Wang L, Mueller C, Humphries M, Spencer LT, Rouhani F, Conlon TJ, Calcedo R, Betts MR, Spencer C, Byrne BJ, Wilson JM, Flotte TR. Brantly ML, et al. Among authors: byrne bj. Proc Natl Acad Sci U S A. 2009 Sep 22;106(38):16363-8. doi: 10.1073/pnas.0904514106. Epub 2009 Aug 12. Proc Natl Acad Sci U S A. 2009. PMID: 19706466 Free PMC article. Clinical Trial.
280 results