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 | 181 | 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 | 134 | 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 | 108 | 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 | 94 | 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 | 89 | 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 | 57 | 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 | 53 | 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 | 44 | 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 | 34 | 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 | 30 | 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 | 28 | 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 | 16 | 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 |
Modulation of Brain Cholesterol Metabolism through CYP46A1 Overexpression for Rett Syndrome E Audouard, N Khefif, B Gillet-Legrand, F Nobilleau, O Bouazizi, S Stanga, ... Pharmaceutics 16 (6), 756, 2024 | 2 | 2024 |
Bioelectronic cell-based device provides a strategy for the treatment of the experimental model of multiple sclerosis E Audouard, F Michel, V Pierroz, T Kim, L Rousselot, B Gillet-Legrand, ... Journal of Controlled Release 352, 994-1008, 2022 | 2 | 2022 |
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 |