Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy

Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model

Bourdon A, François V, Zhang L, Lafoux A, Fraysse B, Toumaniantz G, Larcher T, Girard T, Ledevin M, Lebreton C, Hivonnait A, Creismeas A, Allais M, Marie B, Guguin J, Blouin V, Remy S, Anegon I, Huchet C, Malerba A, Kao B, Le Heron A, Moullier P, Dickson G, Popplewell L, Adjali O, Montanaro F, Le Guiner C. Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model. Gene Ther. 2022 Sep;29(9):520-535. doi: 10.1038/s41434-022-00317-6. Epub 2022 Feb 1. PMID: 35105949

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Date de modification : 26 janvier 2024 | Date de création : 22 mars 2017 | Rédaction : -