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Items: 15


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.

Skelet Muscle. 2018 Apr 27;8(1):15. doi: 10.1186/s13395-018-0161-2.


RFX1 and RFX3 Transcription Factors Interact with the D Sequence of Adeno-Associated Virus Inverted Terminal Repeat and Regulate AAV Transduction.

Julien L, Chassagne J, Peccate C, Lorain S, Piétri-Rouxel F, Danos O, Benkhelifa-Ziyyat S.

Sci Rep. 2018 Jan 9;8(1):210. doi: 10.1038/s41598-017-18604-3.


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.

EMBO Mol Med. 2018 Feb;10(2):239-253. doi: 10.15252/emmm.201707988.


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.

Hum Mol Genet. 2016 Aug 15;25(16):3555-3563. doi: 10.1093/hmg/ddw201. Epub 2016 Jul 4.


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.

Hum Gene Ther. 2016 Sep;27(9):712-26. doi: 10.1089/hum.2016.041. Epub 2016 Jun 8.


Abnormal splicing switch of DMD's penultimate exon compromises muscle fibre maintenance in myotonic dystrophy.

Rau F, Lainé J, Ramanoudjame L, Ferry A, Arandel L, Delalande O, Jollet A, Dingli F, Lee KY, Peccate C, Lorain S, Kabashi E, Athanasopoulos T, Koo T, Loew D, Swanson MS, Le Rumeur E, Dickson G, Allamand V, Marie J, Furling D.

Nat Commun. 2015 May 28;6:7205. doi: 10.1038/ncomms8205.


Dysferlin rescue by spliceosome-mediated pre-mRNA trans-splicing targeting introns harbouring weakly defined 3' splice sites.

Philippi S, Lorain S, Beley C, Peccate C, Précigout G, Spuler S, Garcia L.

Hum Mol Genet. 2015 Jul 15;24(14):4049-60. doi: 10.1093/hmg/ddv141. Epub 2015 Apr 22.


Repair of rhodopsin mRNA by spliceosome-mediated RNA trans-splicing: a new approach for autosomal dominant retinitis pigmentosa.

Berger A, Lorain S, Joséphine C, Desrosiers M, Peccate C, Voit T, Garcia L, Sahel JA, Bemelmans AP.

Mol Ther. 2015 May;23(5):918-930. doi: 10.1038/mt.2015.11. Epub 2015 Jan 26.


Intrinsic transgene immunogenicity gears CD8(+) T-cell priming after rAAV-mediated muscle gene transfer.

Carpentier M, Lorain S, Chappert P, Lalfer M, Hardet R, Urbain D, Peccate C, Adriouch S, Garcia L, Davoust J, Gross DA.

Mol Ther. 2015 Apr;23(4):697-706. doi: 10.1038/mt.2014.235. Epub 2014 Dec 10.


Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches.

Lorain S, Peccate C, Le Hir M, Griffith G, Philippi S, Précigout G, Mamchaoui K, Jollet A, Voit T, Garcia L.

Nucleic Acids Res. 2013 Sep;41(17):8391-402. doi: 10.1093/nar/gkt621. Epub 2013 Jul 16.


AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy.

Le Hir M, Goyenvalle A, Peccate C, Précigout G, Davies KE, Voit T, Garcia L, Lorain S.

Mol Ther. 2013 Aug;21(8):1551-8. doi: 10.1038/mt.2013.121. Epub 2013 Jun 11.


Exon exchange approach to repair Duchenne dystrophin transcripts.

Lorain S, Peccate C, Le Hir M, Garcia L.

PLoS One. 2010 May 28;5(5):e10894. doi: 10.1371/journal.pone.0010894.


Multitissular involvement in a family with LMNA and EMD mutations: Role of digenic mechanism?

Ben Yaou R, Toutain A, Arimura T, Demay L, Massart C, Peccate C, Muchir A, Llense S, Deburgrave N, Leturcq F, Litim KE, Rahmoun-Chiali N, Richard P, Babuty D, Récan-Budiartha D, Bonne G.

Neurology. 2007 May 29;68(22):1883-94.


Protein- and mRNA-based phenotype-genotype correlations in DMD/BMD with point mutations and molecular basis for BMD with nonsense and frameshift mutations in the DMD gene.

Deburgrave N, Daoud F, Llense S, Barbot JC, Récan D, Peccate C, Burghes AH, Béroud C, Garcia L, Kaplan JC, Chelly J, Leturcq F.

Hum Mutat. 2007 Feb;28(2):183-95.


Dystrophinopathy caused by mid-intronic substitutions activating cryptic exons in the DMD gene.

Béroud C, Carrié A, Beldjord C, Deburgrave N, Llense S, Carelle N, Peccate C, Cuisset JM, Pandit F, Carré-Pigeon F, Mayer M, Bellance R, Récan D, Chelly J, Kaplan JC, Leturcq F.

Neuromuscul Disord. 2004 Jan;14(1):10-8.


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