| Intraneuronal aggregation of the β-CTF fragment of APP (C99) induces Aβ-independent lysosomal-autophagic pathology I Lauritzen, R Pardossi-Piquard, A Bourgeois, S Pagnotta, MG Biferi, ... Acta neuropathologica 132, 257-276, 2016 | 202 | 2016 |
| Estrogen-mediated downregulation of AIRE influences sexual dimorphism in autoimmune diseases N Dragin, J Bismuth, G Cizeron-Clairac, MG Biferi, C Berthault, A Serraf, ... The Journal of clinical investigation 126 (4), 1525-1537, 2016 | 202 | 2016 |
| Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase F Puzzo, P Colella, MG Biferi, D Bali, NK Paulk, P Vidal, F Collaud, ... Science translational medicine 9 (418), eaam6375, 2017 | 123 | 2017 |
| Critical requirement for cell cycle inhibitors in sustaining nonproliferative states D Pajalunga, A Mazzola, AM Salzano, MG Biferi, G De Luca, M Crescenzi The Journal of cell biology 176 (6), 807, 2007 | 106 | 2007 |
| Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice Y Tanguy, MG Biferi, A Besse, S Astord, M Cohen-Tannoudji, T Marais, ... Frontiers in molecular neuroscience 8, 36, 2015 | 98 | 2015 |
| Gene therapy for ALS—a perspective M Cappella, C Ciotti, M Cohen-Tannoudji, MG Biferi International journal of molecular sciences 20 (18), 4388, 2019 | 97 | 2019 |
| A new AAV10-U7-mediated gene therapy prolongs survival and restores function in an ALS mouse model MG Biferi, M Cohen-Tannoudji, A Cappelletto, B Giroux, M Roda, S Astord, ... Molecular Therapy 25 (9), 2038-2052, 2017 | 76 | 2017 |
| Cofilin-1 phosphorylation catalyzed by ERK1/2 alters cardiac actin dynamics in dilated cardiomyopathy caused by lamin A/C gene mutation M Chatzifrangkeskou, D Yadin, T Marais, S Chardonnet, ... Human Molecular Genetics 27 (17), 3060-3078, 2018 | 58 | 2018 |
| β-Amyloid precursor protein intracellular domain controls mitochondrial function by modulating phosphatase and tensin homolog–induced kinase 1 transcription in cells and in … T Goiran, E Duplan, M Chami, A Bourgeois, W El Manaa, L Rouland, ... Biological psychiatry 83 (5), 416-427, 2018 | 55 | 2018 |
| AAV9-mediated expression of SMN restricted to neurons does not rescue the spinal muscular atrophy phenotype in mice A Besse, S Astord, T Marais, M Roda, B Giroux, FX Lejeune, F Relaix, ... Molecular Therapy 28 (8), 1887-1901, 2020 | 39 | 2020 |
| The identification of novel biomarkers is required to improve adult SMA patient stratification, diagnosis and treatment P Smeriglio, P Langard, G Querin, MG Biferi Journal of personalized medicine 10 (3), 75, 2020 | 38 | 2020 |
| Beyond the traditional clinical trials for amyotrophic lateral sclerosis and the future impact of gene therapy M Cappella, PF Pradat, G Querin, MG Biferi Journal of Neuromuscular Diseases 8 (1), 25-38, 2021 | 35 | 2021 |
| Targeting γ-secretase triggers the selective enrichment of oligomeric APP-CTFs in brain extracellular vesicles from Alzheimer cell and mouse models I Lauritzen, A Bécot, A Bourgeois, R Pardossi-Piquard, MG Biferi, ... Translational neurodegeneration 8, 1-17, 2019 | 35 | 2019 |
| Biomarkers for C9orf7-ALS in Symptomatic and Pre-symptomatic Patients: State-of-the-art in the New Era of Clinical Trials G Querin, MG Biferi, PF Pradat Journal of neuromuscular diseases 9 (1), 25-37, 2022 | 19 | 2022 |
| Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects P Colella, P Sellier, MJ Gomez, MG Biferi, G Tanniou, N Guerchet, ... EBioMedicine 61, 2020 | 18 | 2020 |
| Systemic treatment of Fabry disease using a novel AAV9 vector expressing α-galactosidase A MG Biferi, M Cohen-Tannoudji, A García-Silva, O Souto-Rodríguez, ... Molecular Therapy-Methods & Clinical Development 20, 1-17, 2021 | 14 | 2021 |
| Lentiviral vector-mediated overexpression of mutant ataxin-7 recapitulates SCA7 pathology and promotes accumulation of the FUS/TLS and MBNL1 RNA-binding proteins S Alves, T Marais, MG Biferi, D Furling, M Marinello, K El Hachimi, ... Molecular Neurodegeneration 11, 1-20, 2016 | 13 | 2016 |
| Promoterless gene targeting approach combined to CRISPR/Cas9 efficiently corrects hemophilia B phenotype in neonatal mice M Lisjak, A De Caneva, T Marais, E Barbon, MG Biferi, F Porro, A Barzel, ... Frontiers in Genome Editing 4, 785698, 2022 | 12 | 2022 |
| Recombinant adeno-associated viral vectors expressing human coagulation FIX-E456H variant in hemophilia B mice S Le Quellec, AP Dane, E Barbon, JC Bordet, F Mingozzi, Y Dargaud, ... Thrombosis and Haemostasis 119 (12), 1956-1967, 2019 | 11 | 2019 |
| Proliferation of multiple cell types in the skeletal muscle tissue elicited by acute p21 suppression MG Biferi, C Nicoletti, G Falcone, EMR Puggioni, N Passaro, A Mazzola, ... Molecular Therapy 23 (5), 885-895, 2015 | 11 | 2015 |
