Adeno-associated virus pseudotype 5 vector improves gene transfer in arthritic joints F Apparailly, M Khoury, MJB Vervoordeldonk, J Adriaansen, E Gicquel, ... Human gene therapy 16 (4), 426-434, 2005 | 96 | 2005 |
Safety and efficacy of AAV-mediated calpain 3 gene transfer in a mouse model of limb-girdle muscular dystrophy type 2A M Bartoli, C Roudaut, S Martin, F Fougerousse, L Suel, J Poupiot, ... Molecular Therapy 13 (2), 250-259, 2006 | 85 | 2006 |
NF‐NF‐κBB‐dependent expression of the antiapoptotic factor c‐FLIP is regulated by calpain 3, the protein involved in limb‐girdle muscular dystrophy type 2A B Benayoun, S Baghdiguian, A Lajmanovich, M Bartoli, N Daniele, ... The FASEB Journal 22 (5), 1521-1529, 2008 | 78 | 2008 |
Mannosidase I inhibition rescues the human α-sarcoglycan R77C recurrent mutation M Bartoli, E Gicquel, L Barrault, T Soheili, M Malissen, B Malissen, ... Human molecular genetics 17 (9), 1214-1221, 2008 | 73 | 2008 |
Phenotypic correction of α-sarcoglycan deficiency by intra-arterial injection of a muscle-specific serotype 1 rAAV vector F Fougerousse, M Bartoli, J Poupiot, L Arandel, M Durand, N Guerchet, ... Molecular Therapy 15 (1), 53-61, 2007 | 73 | 2007 |
Rescue of sarcoglycan mutations by inhibition of endoplasmic reticulum quality control is associated with minimal structural modifications T Soheili, E Gicquel, J Poupiot, L N'Guyen, F Le Roy, M Bartoli, I Richard Human mutation 33 (2), 429-439, 2012 | 50 | 2012 |
A human skeletal muscle interactome centered on proteins involved in muscular dystrophies: LGMD interactome G Blandin, S Marchand, K Charton, N Danièle, E Gicquel, JB Boucheteil, ... Skeletal muscle 3, 1-19, 2013 | 49 | 2013 |
AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression E Gicquel, N Maizonnier, SJ Foltz, WJ Martin, N Bourg, F Svinartchouk, ... Human molecular genetics 26 (10), 1952-1965, 2017 | 45 | 2017 |
Removal of the calpain 3 protease reverses the myopathology in a mouse model for titinopathies K Charton, N Daniele, A Vihola, C Roudaut, E Gicquel, F Monjaret, ... Human Molecular Genetics 19 (23), 4608-4624, 2010 | 43 | 2010 |
Assessing dystrophies and other muscle diseases at the nanometer scale by atomic force microscopy RW Van Zwieten, S Puttini, M Lekka, G Witz, E Gicquel-Zouida, I Richard, ... Nanomedicine 9 (4), 393-406, 2014 | 37 | 2014 |
The Phenotype of Dysferlin-Deficient Mice Is Not Rescued by Adeno-Associated Virus–Mediated Transfer of Anoctamin 5 F Monjaret, L Suel-Petat, N Bourg-Alibert, A Vihola, S Marchand, ... Human gene therapy Clinical development 24 (2), 65-76, 2013 | 20 | 2013 |
216th ENMC international workshop: clinical readiness in FKRP related myopathies January 15–17, 2016 Naarden, The Netherlands I Richard, JP Laurent, S Cirak, J Vissing, S Brown, K Campbell, E Gicquel, ... Neuromuscular Disorders 26 (10), 717-724, 2016 | 17 | 2016 |
Muscle‐specific, liver‐detargeted adeno‐associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa−/− mice P Sellier, P Vidal, B Bertin, E Gicquel, E Bertil‐Froidevaux, C Georger, ... Journal of Inherited Metabolic Disease 47 (1), 119-134, 2024 | 11 | 2024 |
Functional and cellular localization diversity associated with Fukutin‐related protein patient genetic variants SF Henriques, E Gicquel, J Marsolier, I Richard Human Mutation 40 (10), 1874-1885, 2019 | 9 | 2019 |
Assessing dystrophies and other muscle diseases at the nanometer scale by atomic force microscopy V Zwieten, W Ruthger, S Puttini, M Lekka, G Witz, E Gicquel-Zouida, ... Nanomedicine 9 (4), 393-406, 2014 | 8 | 2014 |
Dlk1-Dio3 cluster miRNAs regulate mitochondrial functions in the dystrophic muscle in Duchenne muscular dystrophy AV Hong, N Bourg, P Sanatine, J Poupiot, K Charton, E Gicquel, ... Life Science Alliance 6 (1), 2023 | 7 | 2023 |
Crispr-cas9 ko cell line generation and development of a cell-based potency assay for raav-fkrp gene therapy M Geoffroy, L Pili, V Buffa, M Caroff, A Bigot, E Gicquel, G Rouby, ... Cells 12 (20), 2444, 2023 | 3 | 2023 |
Skeletal muscle cells derived from induced pluripotent stem cells: A platform for limb girdle muscular dystrophies C Bruge, M Geoffroy, M Benabides, E Pellier, E Gicquel, J Dhiab, L Hoch, ... Biomedicines 10 (6), 1428, 2022 | 3 | 2022 |
Hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced liver tropism I Richard, E Gicquel, F Mingozzi US Patent 12,037,362, 2024 | 1 | 2024 |
Synthetic polynucleotides encoding a human FKRP protein I Richard, E Gicquel-zouida, W Lostal US Patent 11,596,698, 2023 | 1 | 2023 |