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Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice.
Dominguez E, Marais T, Chatauret N, Benkhelifa-Ziyyat S, Duque S, Ravassard P, Carcenac R, Astord S, Pereira de Moura A, Voit T, Barkats M. Dominguez E, et al. Among authors: voit t. Hum Mol Genet. 2011 Feb 15;20(4):681-93. doi: 10.1093/hmg/ddq514. Epub 2010 Nov 30. Hum Mol Genet. 2011. PMID: 21118896
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: voit t. 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
Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.
Armbruster N, Lattanzi A, Jeavons M, Van Wittenberghe L, Gjata B, Marais T, Martin S, Vignaud A, Voit T, Mavilio F, Barkats M, Buj-Bello A. Armbruster N, et al. Among authors: voit t. Mol Ther Methods Clin Dev. 2016 Sep 14;3:16060. doi: 10.1038/mtm.2016.60. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 27652289 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. Among authors: voit t. 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
Serum proteomic profiling reveals fragments of MYOM3 as potential biomarkers for monitoring the outcome of therapeutic interventions in muscular dystrophies.
Rouillon J, Poupiot J, Zocevic A, Amor F, Léger T, Garcia C, Camadro JM, Wong B, Pinilla R, Cosette J, Coenen-Stass AM, Mcclorey G, Roberts TC, Wood MJ, Servais L, Udd B, Voit T, Richard I, Svinartchouk F. Rouillon J, et al. Among authors: voit t. Hum Mol Genet. 2015 Sep 1;24(17):4916-32. doi: 10.1093/hmg/ddv214. Epub 2015 Jun 9. Hum Mol Genet. 2015. PMID: 26060189 Free PMC article.
317 results