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Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders.
El Andari J, Renaud-Gabardos E, Tulalamba W, Weinmann J, Mangin L, Pham QH, Hille S, Bennett A, Attebi E, Bourges E, Leborgne C, Guerchet N, Fakhiri J, Krämer C, Wiedtke E, McKenna R, Guianvarc'h L, Toueille M, Ronzitti G, Hebben M, Mingozzi F, VandenDriessche T, Agbandje-McKenna M, Müller OJ, Chuah MK, Buj-Bello A, Grimm D. El Andari J, et al. Among authors: kramer c. Sci Adv. 2022 Sep 23;8(38):eabn4704. doi: 10.1126/sciadv.abn4704. Epub 2022 Sep 21. Sci Adv. 2022. PMID: 36129972 Free PMC article.
Striated muscle-specific base editing enables correction of mutations causing dilated cardiomyopathy.
Grosch M, Schraft L, Chan A, Küchenhoff L, Rapti K, Ferreira AM, Kornienko J, Li S, Radke MH, Krämer C, Clauder-Münster S, Perlas E, Backs J, Gotthardt M, Dieterich C, van den Hoogenhof MMG, Grimm D, Steinmetz LM. Grosch M, et al. Among authors: kramer c. Nat Commun. 2023 Jun 22;14(1):3714. doi: 10.1038/s41467-023-39352-1. Nat Commun. 2023. PMID: 37349314 Free PMC article.