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Optogenetic modeling of human neuromuscular circuits in Duchenne muscular dystrophy with CRISPR and pharmacological corrections.
Paredes-Redondo A, Harley P, Maniati E, Ryan D, Louzada S, Meng J, Kowala A, Fu B, Yang F, Liu P, Marino S, Pourquié O, Muntoni F, Wang J, Lieberam I, Lin YY. Paredes-Redondo A, et al. Among authors: kowala a. Sci Adv. 2021 Sep 10;7(37):eabi8787. doi: 10.1126/sciadv.abi8787. Epub 2021 Sep 10. Sci Adv. 2021. PMID: 34516770 Free PMC article.
Investigating the role of dystrophin isoform deficiency in motor function in Duchenne muscular dystrophy.
Chesshyre M, Ridout D, Hashimoto Y, Ookubo Y, Torelli S, Maresh K, Ricotti V, Abbott L, Gupta VA, Main M, Ferrari G, Kowala A, Lin YY, Tedesco FS, Scoto M, Baranello G, Manzur A, Aoki Y, Muntoni F. Chesshyre M, et al. Among authors: kowala a. J Cachexia Sarcopenia Muscle. 2022 Apr;13(2):1360-1372. doi: 10.1002/jcsm.12914. Epub 2022 Jan 26. J Cachexia Sarcopenia Muscle. 2022. PMID: 35083887 Free PMC article.
CRISPR-mediated correction of skeletal muscle Ca2+ handling in a novel DMD patient-derived pluripotent stem cell model.
Morera C, Kim J, Paredes-Redondo A, Nobles M, Rybin D, Moccia R, Kowala A, Meng J, Garren S, Liu P, Morgan JE, Muntoni F, Christoforou N, Owens J, Tinker A, Lin YY. Morera C, et al. Among authors: kowala a. Neuromuscul Disord. 2022 Dec;32(11-12):908-922. doi: 10.1016/j.nmd.2022.10.007. Epub 2022 Nov 1. Neuromuscul Disord. 2022. PMID: 36418198 Free article.