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CYP46A1, the rate-limiting enzyme for cholesterol degradation, is neuroprotective in Huntington's disease

Brain (London, England : 1878), 2016-03, Vol.139 (Pt 3), p.953-970

2016

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Clinical Gene Therapy for Neurodegenerative Diseases: Past, Present, and Future

Human gene therapy, 2017-11, Vol.28 (11), p.988-1003

2017

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Rapid and Complete Reversal of Sensory Ataxia by Gene Therapy in a Novel Model of Friedreich Ataxia

Molecular therapy, 2018-08, Vol.26 (8), p.1940-1952

2018

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Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice

Frontiers in molecular neuroscience, 2021-05, Vol.14, p.677895-677895

2021

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The Challenge of Gene Therapy for Neurological Diseases: Strategies and Tools to Achieve Efficient Delivery to the Central Nervous System

Human gene therapy, 2021-04, Vol.32 (7-8), p.349-374

2021

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AAV‐delivered diacylglycerol kinase DGKk achieves long‐term rescue of fragile X syndrome mouse model

EMBO molecular medicine, 2022-05, Vol.14 (5), p.e14649-n/a

2022

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Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice

Human gene therapy, 2012-08, Vol.23 (8), p.903-914

2012

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Gene therapy for neurodegenerative disorders in children: dreams and realities

Archives de pédiatrie : organe officiel de la Société française de pédiatrie, 2023-11, Vol.30 (8), p.8-8S40

2023

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Niacin‐mediated Tace activation ameliorates CMT neuropathies with focal hypermyelination

EMBO molecular medicine, 2016-12, Vol.8 (12), p.1438-1454

2016

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SCA7 Mouse Cerebellar Pathology Reveals Preferential Downregulation of Key Purkinje Cell-Identity Genes and Shared Disease Signature with SCA1 and SCA2

The Journal of neuroscience, 2021-06, Vol.41 (22), p.4910-4936

2021

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DDIT4/REDD1/RTP801 is a novel negative regulator of Schwann cell myelination

The Journal of neuroscience, 2013-09, Vol.33 (38), p.15295-15305

2013

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Modulation of Brain Cholesterol Metabolism through CYP46A1 Overexpression for Rett Syndrome

Pharmaceutics, 2024-06, Vol.16 (6), p.756

2024

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Optimizing PCR for Mouse Genotyping: Recommendations for Reliable, Rapid, Cost Effective, Robust and Adaptable to High‐Throughput Genotyping Protocol for Any Type of Mutation

Current protocols in mouse biology, 2019-12, Vol.9 (4), p.e65-n/a

2019

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Dose-response evaluation of intravenous gene therapy in a symptomatic mouse model of metachromatic leukodystrophy

Molecular therapy. Methods & clinical development, 2024-06, Vol.32 (2), p.101248-101248, Article 101248

2024

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Cholesterol redistribution triggered by CYP46A1 gene therapy improves major hallmarks of Niemann-Pick type C disease but is not sufficient to halt neurodegeneration

Biochimica et biophysica acta. Molecular basis of disease, 2024-03, Vol.1870 (3), p.166993-166993, Article 166993

2024

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Optimized Protocol for Subcutaneous Implantation of Encapsulated Cells Device and Evaluation of Biocompatibility

Frontiers in bioengineering and biotechnology, 2021-06, Vol.9, p.620967-620967

2021

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Nouvelles approches de la thérapie génique

Revue neurologique, 2022-04, Vol.178, p.S175-S175

Piguet, Françoise

2022

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Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate

Human molecular genetics, 2010-01, Vol.19 (1), p.147-158

2010

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Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates

Human gene therapy. Clinical development, 2015-06, Vol.26 (2), p.113-124

2015

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Bioelectronic cell-based device provides a strategy for the treatment of the experimental model of multiple sclerosis

Journal of controlled release, 2022-12, Vol.352, p.994-1008

2022

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Intravenous gene therapy using AAVPHP.eB for metachromatic leukodystrophy

Molecular genetics and metabolism, 2024-02, Vol.141 (2), p.107758, Article 107758

2024

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Cell-based device provides effective therapeutic strategy to treat metachromatic leukodystrophy

Molecular genetics and metabolism, 2024-02, Vol.141 (2), p.107759, Article 107759

2024

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Development and validation of a novel adeno-associated viral gene therapy for mucopolysaccharidosis type IIIB (MPS IIIB)

Molecular genetics and metabolism, 2023-02, Vol.138 (2), p.107019, Article 107019

2023

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Development and validation of a novel AAV gene therapy for mucopolysaccharidosis type IIIB in large animal

Molecular genetics and metabolism, 2024-02, Vol.141 (2), p.107768, Article 107768

2024

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Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain

Molecular therapy. Methods & clinical development, 2019-09, Vol.14, p.237-251

2019

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CYP46A1, the rate-limiting enzyme for cholesterol degradation, is neuroprotective in Huntington's disease

Clinical Gene Therapy for Neurodegenerative Diseases: Past, Present, and Future

Rapid and Complete Reversal of Sensory Ataxia by Gene Therapy in a Novel Model of Friedreich Ataxia

Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice

The Challenge of Gene Therapy for Neurological Diseases: Strategies and Tools to Achieve Efficient Delivery to the Central Nervous System

AAV‐delivered diacylglycerol kinase DGKk achieves long‐term rescue of fragile X syndrome mouse model

Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice

Gene therapy for neurodegenerative disorders in children: dreams and realities

Niacin‐mediated Tace activation ameliorates CMT neuropathies with focal hypermyelination

SCA7 Mouse Cerebellar Pathology Reveals Preferential Downregulation of Key Purkinje Cell-Identity Genes and Shared Disease Signature with SCA1 and SCA2

DDIT4/REDD1/RTP801 is a novel negative regulator of Schwann cell myelination

Modulation of Brain Cholesterol Metabolism through CYP46A1 Overexpression for Rett Syndrome

Optimizing PCR for Mouse Genotyping: Recommendations for Reliable, Rapid, Cost Effective, Robust and Adaptable to High‐Throughput Genotyping Protocol for Any Type of Mutation

Dose-response evaluation of intravenous gene therapy in a symptomatic mouse model of metachromatic leukodystrophy

Cholesterol redistribution triggered by CYP46A1 gene therapy improves major hallmarks of Niemann-Pick type C disease but is not sufficient to halt neurodegeneration

Optimized Protocol for Subcutaneous Implantation of Encapsulated Cells Device and Evaluation of Biocompatibility

Nouvelles approches de la thérapie génique

Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate

Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates

Bioelectronic cell-based device provides a strategy for the treatment of the experimental model of multiple sclerosis

Intravenous gene therapy using AAVPHP.eB for metachromatic leukodystrophy

Cell-based device provides effective therapeutic strategy to treat metachromatic leukodystrophy

Development and validation of a novel adeno-associated viral gene therapy for mucopolysaccharidosis type IIIB (MPS IIIB)

Development and validation of a novel AAV gene therapy for mucopolysaccharidosis type IIIB in large animal

Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain

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