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2006 1
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Page 1
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. Among authors: lorain s. EMBO Mol Med. 2017 May;9(5):545-557. doi: 10.15252/emmm.201607199. EMBO Mol Med. 2017. PMID: 28289078 Free PMC article. Review.
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.
Gene Therapy via Trans-Splicing for LMNA-Related Congenital Muscular Dystrophy.
Azibani F, Brull A, Arandel L, Beuvin M, Nelson I, Jollet A, Ziat E, Prudhon B, Benkhelifa-Ziyyat S, Bitoun M, Lorain S, Bonne G, Bertrand AT. Azibani F, et al. Among authors: lorain s. Mol Ther Nucleic Acids. 2018 Mar 2;10:376-386. doi: 10.1016/j.omtn.2017.12.012. Epub 2017 Dec 30. Mol Ther Nucleic Acids. 2018. PMID: 29499949 Free PMC article.
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
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.
Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice.
Gedicke-Hornung C, Behrens-Gawlik V, Reischmann S, Geertz B, Stimpel D, Weinberger F, Schlossarek S, Précigout G, Braren I, Eschenhagen T, Mearini G, Lorain S, Voit T, Dreyfus PA, Garcia L, Carrier L. Gedicke-Hornung C, et al. Among authors: lorain s. EMBO Mol Med. 2013 Jul;5(7):1128-45. doi: 10.1002/emmm.201202168. Epub 2013 May 29. EMBO Mol Med. 2013. PMID: 23716398 Free PMC article.
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