Nicolas Guerchet

Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer

William Lostal, Marc Bartoli, Nathalie Bourg et al. · Human Molecular Genetics · 2010 · 170 citations

Medicine, Genetics, Molecular Biology +17

Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders

Jihad El Andari, Edith Renaud-Gabardos, Warut Tulalamba et al. · Science Advances · 2022 · 92 citations · Full text

Pharmacy, Bioengineering, Biotechnology +13

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

Pasqualina Colella, Pauline Sellier, Helena Costa Verdera et al. · Molecular Therapy — Methods & Clinical Development · 2018 · 73 citations · Full text

Pharmacy, Genetics, Molecular Genetics +16

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

Françoise Fougerousse, Marc Bartoli, Jérôme Poupiot et al. · Molecular Therapy · 2006 · 65 citations · Full text

Medicine, Molecular Biology, Molecular Genetics +15

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects

Pasqualina Colella, Pauline Sellier, Manuel J. Gómez et al. · EBioMedicine · 2020 · 27 citations · Full text

Medicine, Molecular Biology, Molecular Genetics +14