| CYP46A1, the rate-limiting enzyme for cholesterol degradation, is neuroprotective in Huntington’s disease L Boussicault, S Alves, A Lamazière, A Planques, N Heck, L Moumné, ... Brain 139 (3), 953-970, 2016 | 176 | 2016 |
| Clinical gene therapy for neurodegenerative diseases: past, present, and future F Piguet, S Alves, N Cartier Human gene therapy 28 (11), 988-1003, 2017 | 132 | 2017 |
| Rapid and complete reversal of sensory ataxia by gene therapy in a novel model of Friedreich ataxia F Piguet, C de Montigny, N Vaucamps, L Reutenauer, A Eisenmann, ... Molecular Therapy 26 (8), 1940-1952, 2018 | 100 | 2018 |
| Correction of brain oligodendrocytes by AAVrh. 10 intracerebral gene therapy in metachromatic leukodystrophy mice F Piguet, D Sondhi, M Piraud, F Fouquet, NR Hackett, O Ahouansou, ... Human gene therapy 23 (8), 903-914, 2012 | 92 | 2012 |
| Intracerebral gene therapy using AAVrh. 10-hARSA recombinant vector to treat patients with early-onset forms of metachromatic leukodystrophy: preclinical feasibility and safety … M Zerah, F Piguet, MA Colle, S Raoul, JY Deschamps, J Deniaud, ... Human Gene Therapy Clinical Development 26 (2), 113-124, 2015 | 87 | 2015 |
| Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate MA Colle, F Piguet, L Bertrand, S Raoul, I Bieche, L Dubreil, D Sloothaak, ... Human molecular genetics 19 (1), 147-158, 2010 | 84 | 2010 |
| DDIT4/REDD1/RTP801 is a novel negative regulator of Schwann cell myelination R Noseda, S Belin, F Piguet, I Vaccari, S Scarlino, P Brambilla, ... Journal of Neuroscience 33 (38), 15295-15305, 2013 | 56 | 2013 |
| Niacin‐mediated Tace activation ameliorates CMT neuropathies with focal hypermyelination A Bolino, F Piguet, V Alberizzi, M Pellegatta, C Rivellini, ... EMBO molecular medicine 8 (12), 1438-1454, 2016 | 51 | 2016 |
| Real-time monitoring of exosome enveloped-AAV spreading by endomicroscopy approach: a new tool for gene delivery in the brain NS Orefice, B Souchet, J Braudeau, S Alves, F Piguet, F Collaud, ... Molecular Therapy Methods & Clinical Development 14, 237-251, 2019 | 41 | 2019 |
| SCA7 mouse cerebellar pathology reveals preferential downregulation of key Purkinje cell-identity genes and shared disease signature with SCA1 and SCA2 A Niewiadomska-Cimicka, F Doussau, JB Perot, MJ Roux, C Keime, ... Journal of Neuroscience 41 (22), 4910-4936, 2021 | 32 | 2021 |
| Optimizing PCR for Mouse Genotyping: Recommendations for Reliable, Rapid, Cost Effective, Robust and Adaptable to High‐Throughput Genotyping Protocol for Any Type of Mutation S Jacquot, N Chartoire, F Piguet, Y Hérault, G Pavlovic Current Protocols in Mouse Biology 9 (4), e65, 2019 | 29 | 2019 |
| The challenge of gene therapy for neurological diseases: strategies and tools to achieve efficient delivery to the central nervous system F Piguet, T de Saint Denis, E Audouard, K Beccaria, A André, G Wurtz, ... Human Gene Therapy 32 (7-8), 349-374, 2021 | 25 | 2021 |
| Complete correction of brain and spinal cord pathology in metachromatic leukodystrophy mice E Audouard, V Oger, B Meha, N Cartier, C Sevin, F Piguet Frontiers in Molecular Neuroscience 14, 677895, 2021 | 15 | 2021 |
| AAV‐delivered diacylglycerol kinase DGKk achieves long‐term rescue of fragile X syndrome mouse model K Habbas, O Cakil, B Zámbó, R Tabet, F Riet, D Dembele, JL Mandel, ... EMBO Molecular Medicine 14 (5), e14649, 2022 | 10 | 2022 |
| Optimized protocol for subcutaneous implantation of encapsulated cells device and evaluation of biocompatibility E Audouard, L Rousselot, M Folcher, N Cartier, F Piguet Frontiers in bioengineering and biotechnology 9, 620967, 2021 | 7 | 2021 |
| Restoring neuronal cholesterol efficiently rescues ALS mouse model F Piguet, G Wurtz, E Audouard, BG Legrand, N Cartier-Lacave HUMAN GENE THERAPY 30 (11), A94-A94, 2019 | 4 | 2019 |
| Methods and pharmaceutical composition for the treatment and the prevention of neurological phenotype associated with Friedreich ataxia H Puccio, F Piguet US Patent 11,040,113, 2021 | 3 | 2021 |
| O5‐06‐04: AAV‐CYP46A1 BRAIN DELIVERY MITIGATES ALZHEIMER'S DISEASE: FROM MOUSE MODELS TO NON‐HUMAN PRIMATES S Alves, K Michaelsen-Preusse, M Audrain, RA Badin, A Lamazière, ... Alzheimer's & Dementia 14 (7S_Part_31), P1658-P1659, 2018 | 2 | 2018 |
| ESGCT XXV anniversary congress in collaboration with the German Society for Gene Therapy, October 17–20, 2017 Berlin, Germany S Maffioletti Human Gene Therapy 28 (12), A1-A125, 2017 | 2 | 2017 |
| Dose-response evaluation of intravenous gene therapy in a symptomatic mouse model of metachromatic leukodystrophy E Audouard, N Khefif, C Mansat, O Nelcha, EG Banchi, C Lupiet, ... Molecular Therapy Methods & Clinical Development 32 (2), 2024 | 1 | 2024 |
Michel ZERAHProfessor of pediatric neurosurgery在 aphp.fr 的电子邮件经过验证
