Exon Exchange Approach to Repair Duchenne Dystrophin Transcripts

Por Lorain, Stéphanie, Peccate, Cécile, Le Hir, Maëva, Garcia, Luis
Publicado em 2010

AAV Genome Loss From Dystrophic Mouse Muscles During AAV-U7 snRNA-mediated Exon-skipping Therapy

Por Le Hir, Maëva, Goyenvalle, Aurélie, Peccate, Cécile, Précigout, Guillaume, Davies, Kay E, Voit, Thomas, Garcia, Luis, Lorain, Stéphanie
Publicado em 2013

Repair of Rhodopsin mRNA by Spliceosome-Mediated RNA Trans-Splicing: A New Approach for Autosomal Dominant Retinitis Pigmentosa

Por Berger, Adeline, Lorain, Stéphanie, Joséphine, Charlène, Desrosiers, Melissa, Peccate, Cécile, Voit, Thomas, Garcia, Luis, Sahel, José-Alain, Bemelmans, Alexis-Pierre
Publicado no Mol Ther (2015)

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

Por Lorain, Stéphanie, Peccate, Cécile, Le Hir, Maëva, Griffith, Graziella, Philippi, Susanne, Précigout, Guillaume, Mamchaoui, Kamel, Jollet, Arnaud, Voit, Thomas, Garcia, Luis
Publicado em 2013

Intrinsic Transgene Immunogenicity Gears CD8(+) T-cell Priming After rAAV-Mediated Muscle Gene Transfer

Por Carpentier, Maxime, Lorain, Stéphanie, Chappert, Pascal, Lalfer, Mélanie, Hardet, Romain, Urbain, Dominique, Peccate, Cécile, Adriouch, Sahil, Garcia, Luis, Davoust, Jean, Gross, David-Alexandre
Publicado no Mol Ther (2015)

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

Por Rau, Frédérique, Lainé, Jeanne, Ramanoudjame, Laetitita, Ferry, Arnaud, Arandel, Ludovic, Delalande, Olivier, Jollet, Arnaud, Dingli, Florent, Lee, Kuang-Yung, Peccate, Cécile, Lorain, Stéphanie, Kabashi, Edor, Athanasopoulos, Takis, Koo, Taeyoung, Loew, Damarys, Swanson, Maurice S., Le Rumeur, Elisabeth, Dickson, George, Allamand, Valérie, Marie, Joëlle, Furling, Denis
Publicado no Nat Commun (2015)