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Human Mutation|July 4, 2019
Functional and cellular localization diversity associated with Fukutin-related protein patient genetic variantsSara F Henriques, Evelyne Gicquel, Justine Marsolier, et al.
Journal of Neuromuscular Diseases|September 14, 2020
Minimal Consequences of CMAH and DBA/2 Backgrounds on a FKRP Deficient ModelCamille Vaubourg, Evelyne Gicquel, Isabelle Richard, et al.
Medecine Sciences : M/S|November 18, 2024
[Advances and Challenges in Microdystrophin gene therapy for Duchenne Muscular Dystrophy: progress and future directions]Abbass Jaber, Laura Palmieri, Evelyne Gicquel, et al.
Human Mutation|November 19, 2011
Rescue of sarcoglycan mutations by inhibition of endoplasmic reticulum quality control is associated with minimal structural modificationsTayebeh Soheili, Evelyne Gicquel, Jérôme Poupiot, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 14, 2006
Phenotypic correction of alpha-sarcoglycan deficiency by intra-arterial injection of a muscle-specific serotype 1 rAAV vectorFrancoise Fougerousse, Marc Bartoli, Jérôme Poupiot, et al.
Cells|October 27, 2023
CRISPR-Cas9 KO Cell Line Generation and Development of a Cell-Based Potency Assay for rAAV-FKRP Gene TherapyMarine Geoffroy, Louna Pili, Valentina Buffa, et al.
Biomedicines|June 24, 2022
Skeletal Muscle Cells Derived from Induced Pluripotent Stem Cells: A Platform for Limb Girdle Muscular DystrophiesCeline Bruge, Marine Geoffroy, Manon Benabides, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 10, 2017
Phenotypic Correction of α-Sarcoglycan Deficiency by Intra-arterial Injection of a Muscle-specific Serotype 1 rAAV VectorFrancoise Fougerousse, Marc Bartoli, Jérôme Poupiot, et al.
Human Molecular Genetics|September 22, 2010
Removal of the calpain 3 protease reverses the myopathology in a mouse model for titinopathiesKarine Charton, Nathalie Danièle, Anna Vihola, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 18, 2005
Safety and efficacy of AAV-mediated calpain 3 gene transfer in a mouse model of limb-girdle muscular dystrophy type 2AMarc Bartoli, Carinne Roudaut, Samia Martin, et al.
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