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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. Among authors: chamberlain js. 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.
Efficacy and muscle safety assessment of fukutin-related protein gene therapy.
Benasutti H, Maricelli JW, Seto J, Hall J, Halbert C, Wicki J, Heusgen L, Purvis N, Regnier M, Lin DC, Rodgers BD, Chamberlain JS. Benasutti H, et al. Among authors: chamberlain js. Mol Ther Methods Clin Dev. 2023 Jun 4;30:65-80. doi: 10.1016/j.omtm.2023.05.022. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37361354 Free PMC article.
Biomarkers for Duchenne muscular dystrophy progression: impact of age in the mdx tongue spared muscle.
Lorena MDSV, Santos EK, Ferretti R, Gowda GAN, Odom GL, Chamberlain JS, Matsumura CY. Lorena MDSV, et al. Among authors: chamberlain js. Res Sq [Preprint]. 2023 Jun 13:rs.3.rs-3038923. doi: 10.21203/rs.3.rs-3038923/v1. Res Sq. 2023. Update in: Skelet Muscle. 2023 Sep 13;13(1):16. doi: 10.1186/s13395-023-00325-z. PMID: 37398370 Free PMC article. Updated. Preprint.
Enveloped viruses pseudotyped with mammalian myogenic cell fusogens target skeletal muscle for gene delivery.
Hindi SM, Petrany MJ, Greenfeld E, Focke LC, Cramer AAW, Whitt MA, Prasad V, Chamberlain JS, Podbilewicz B, Millay DP. Hindi SM, et al. Among authors: chamberlain js. bioRxiv [Preprint]. 2023 Mar 18:2023.03.17.533157. doi: 10.1101/2023.03.17.533157. bioRxiv. 2023. Update in: Cell. 2023 May 11;186(10):2062-2077.e17. doi: 10.1016/j.cell.2023.03.033. PMID: 36993357 Free PMC article. Updated. Preprint.
249 results