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Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.
Peccate C, Mollard A, Le Hir M, Julien L, McClorey G, Jarmin S, Le Heron A, Dickson G, Benkhelifa-Ziyyat S, Piétri-Rouxel F, Wood MJ, Voit T, Lorain S. Peccate C, et al. Hum Mol Genet. 2016 Aug 15;25(16):3555-3563. doi: 10.1093/hmg/ddw201. Epub 2016 Jul 4. Hum Mol Genet. 2016. PMID: 27378686
DHPR alpha1S subunit controls skeletal muscle mass and morphogenesis.
Piétri-Rouxel F, Gentil C, Vassilopoulos S, Baas D, Mouisel E, Ferry A, Vignaud A, Hourdé C, Marty I, Schaeffer L, Voit T, Garcia L. Piétri-Rouxel F, et al. EMBO J. 2010 Feb 3;29(3):643-54. doi: 10.1038/emboj.2009.366. Epub 2009 Dec 24. EMBO J. 2010. PMID: 20033060 Free PMC article.
Dystrophin Threshold Level Necessary for Normalization of Neuronal Nitric Oxide Synthase, Inducible Nitric Oxide Synthase, and Ryanodine Receptor-Calcium Release Channel Type 1 Nitrosylation in Golden Retriever Muscular Dystrophy Dystrophinopathy.
Gentil C, Le Guiner C, Falcone S, Hogrel JY, Peccate C, Lorain S, Benkhelifa-Ziyyat S, Guigand L, Montus M, Servais L, Voit T, Piétri-Rouxel F. Gentil C, et al. Hum Gene Ther. 2016 Sep;27(9):712-26. doi: 10.1089/hum.2016.041. Epub 2016 Jun 8. Hum Gene Ther. 2016. PMID: 27279388
Delivery is key: lessons learnt from developing splice-switching antisense therapies.
Godfrey C, Desviat LR, Smedsrød B, Piétri-Rouxel F, Denti MA, Disterer P, Lorain S, Nogales-Gadea G, Sardone V, Anwar R, El Andaloussi S, Lehto T, Khoo B, Brolin C, van Roon-Mom WM, Goyenvalle A, Aartsma-Rus A, Arechavala-Gomeza V. Godfrey C, et al. EMBO Mol Med. 2017 May;9(5):545-557. doi: 10.15252/emmm.201607199. EMBO Mol Med. 2017. PMID: 28289078 Free PMC article. Review.
miR-708-5p and miR-34c-5p are involved in nNOS regulation in dystrophic context.
Guilbaud M, Gentil C, Peccate C, Gargaun E, Holtzmann I, Gruszczynski C, Falcone S, Mamchaoui K, Ben Yaou R, Leturcq F, Jeanson-Leh L, Piétri-Rouxel F. Guilbaud M, et al. Skelet Muscle. 2018 Apr 27;8(1):15. doi: 10.1186/s13395-018-0161-2. Skelet Muscle. 2018. PMID: 29703249 Free PMC article.
42 results