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Page 1
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model.
Piepho AB, Lowe J, Cumby LR, Dorn LE, Lake DM, Rastogi N, Gertzen MD, Sturgill SL, Odom GL, Ziolo MT, Accornero F, Chamberlain JS, Rafael-Fortney JA. Piepho AB, et al. Mol Ther Methods Clin Dev. 2023 Feb 9;28:344-354. doi: 10.1016/j.omtm.2023.02.001. eCollection 2023 Mar 9. Mol Ther Methods Clin Dev. 2023. PMID: 36874243 Free PMC article.
A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2.
Flanigan KM, Vetter TA, Simmons TR, Iammarino M, Frair EC, Rinaldi F, Chicoine LG, Harris J, Cheatham JP, Cheatham SL, Boe B, Waldrop MA, Zygmunt DA, Packer D, Martin PT. Flanigan KM, et al. Mol Ther Methods Clin Dev. 2022 Sep 2;27:47-60. doi: 10.1016/j.omtm.2022.08.009. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36186954 Free PMC article.
Short-term treatment of golden retriever muscular dystrophy (GRMD) dogs with rAAVrh74.MHCK7.GALGT2 induces muscle glycosylation and utrophin expression but has no significant effect on muscle strength.
Martin PT, Zygmunt DA, Ashbrook A, Hamilton S, Packer D, Birch SM, Bettis AK, Balog-Alvarez CJ, Guo LJ, Nghiem PP, Kornegay JN. Martin PT, et al. PLoS One. 2021 Mar 26;16(3):e0248721. doi: 10.1371/journal.pone.0248721. eCollection 2021. PLoS One. 2021. PMID: 33770101 Free PMC article.
22 results