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Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice.
Saoudi A, Barberat S, le Coz O, Vacca O, Doisy Caquant M, Tensorer T, Sliwinski E, Garcia L, Muntoni F, Vaillend C, Goyenvalle A. Saoudi A, et al. Among authors: goyenvalle a. Mol Ther Nucleic Acids. 2023 Mar 21;32:173-188. doi: 10.1016/j.omtn.2023.03.009. eCollection 2023 Jun 13. Mol Ther Nucleic Acids. 2023. PMID: 37078061 Free PMC article.
Immortalized skin fibroblasts expressing conditional MyoD as a renewable and reliable source of converted human muscle cells to assess therapeutic strategies for muscular dystrophies: validation of an exon-skipping approach to restore dystrophin in Duchenne muscular dystrophy cells.
Chaouch S, Mouly V, Goyenvalle A, Vulin A, Mamchaoui K, Negroni E, Di Santo J, Butler-Browne G, Torrente Y, Garcia L, Furling D. Chaouch S, et al. Among authors: goyenvalle a. Hum Gene Ther. 2009 Jul;20(7):784-90. doi: 10.1089/hum.2008.163. Hum Gene Ther. 2009. PMID: 19358679
Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping.
Vulin A, Barthélémy I, Goyenvalle A, Thibaud JL, Beley C, Griffith G, Benchaouir R, le Hir M, Unterfinger Y, Lorain S, Dreyfus P, Voit T, Carlier P, Blot S, Garcia L. Vulin A, et al. Among authors: goyenvalle a. Mol Ther. 2012 Nov;20(11):2120-33. doi: 10.1038/mt.2012.181. Epub 2012 Sep 11. Mol Ther. 2012. PMID: 22968479 Free PMC article.
69 results