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Page 1
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. Among authors: lorain s. 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
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. Among authors: lorain s. 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
Allele-specific silencing therapy for Dynamin 2-related dominant centronuclear myopathy.
Trochet D, Prudhon B, Beuvin M, Peccate C, Lorain S, Julien L, Benkhelifa-Ziyyat S, Rabai A, Mamchaoui K, Ferry A, Laporte J, Guicheney P, Vassilopoulos S, Bitoun M. Trochet D, et al. Among authors: lorain s. EMBO Mol Med. 2018 Feb;10(2):239-253. doi: 10.15252/emmm.201707988. EMBO Mol Med. 2018. PMID: 29246969 Free PMC article.
Combined Treatment with Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer-PPMO and AAV-U7 Rescues the Severe DMD Phenotype in Mice.
Forand A, Muchir A, Mougenot N, Sevoz-Couche C, Peccate C, Lemaitre M, Izabelle C, Wood M, Lorain S, Piétri-Rouxel F. Forand A, et al. Among authors: lorain s. Mol Ther Methods Clin Dev. 2020 Mar 17;17:695-708. doi: 10.1016/j.omtm.2020.03.011. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32346547 Free PMC article.
31 results