Safety and Efficacy of AAV-Mediated Calpain 3 Gene Transfer in a Mouse Model of Limb-Girdle Muscular Dystrophy Type 2A

Marc Bartoli, Carinne Roudaut, Samia Martin, +7

Molecular Therapy

MechanobiologyCalpain 3Muscle FunctionMolecular NeuroscienceMolecular Physiology +13

Adeno-Associated Virus Pseudotype 5 Vector Improves Gene Transfer in Arthritic Joints

Florence Apparailly, Maroun Khoury, Margriet J. Vervoordeldonk, +9

Human Gene Therapy

NF‐NF <scp>‐κ</scp> BB‐dependent expression of the antiapoptotic factor c‐FLIP is regulated by calpain 3, the protein involved in limb‐girdle muscular dystrophy type 2A

Béatrice Benayoun, Stephen Baghdiguian, Alicia Lajmanovich, +9

The FASEB Journal

Phenotypic Correction of α-Sarcoglycan Deficiency by Intra-arterial Injection of a Muscle-specific Serotype 1 rAAV Vector

Françoise Fougerousse, Marc Bartoli, Jérôme Poupiot, +6

Molecular Therapy

Mannosidase I inhibition rescues the human α-sarcoglycan R77C recurrent mutation

Marc Bartoli, Evelyne Gicquel, Laetitia Barrault, +8

Human Molecular Genetics

Rescue of sarcoglycan mutations by inhibition of endoplasmic reticulum quality control is associated with minimal structural modifications

Tayebeh Soheili, Evelyne Gicquel, Jérôme Poupiot, +4

Human Mutation

Sarcoglycan MutationsMolecular BiologyCellular PhysiologyMuscle MembraneLocalization Rescue +16

A human skeletal muscle interactome centered on proteins involved in muscular dystrophies: LGMD interactome

Gaëlle Blandin, Sylvie Marchand, Karine Charton, +8

Skeletal Muscle

AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression

Evelyne Gicquel, Natacha Maizonnier, Steven Foltz, +6

Human Molecular Genetics

αDg GlycosylationMuscle FunctionAbnormal GlycosylationImmunologyBiological Therapy +26

Removal of the calpain 3 protease reverses the myopathology in a mouse model for titinopathies

Karine Charton, Nathalie Danièle, Anna Vihola, +7

Human Molecular Genetics

MechanobiologyCalpain 3Molecular PhysiologyDevelopmental BiologySkeletal Muscle +13

Investigation of the Positive Response of Ethyl Acrylate in the Mouse Lymphoma Genotoxicity Assay

Paul J. Ciaccio, Evelyne Gicquel, P. J. O'Neill, +2

Toxicological Sciences

Dna DamageImmunotoxicologyApoptosisCell DeathPathology +17