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Patrick Aubourg

French Institute of Health and Medical Research

167

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7441

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AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A.

2021 Nature Communications
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Elivaldogene Autotemcel (eli-cel, Lenti-D) Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy: Updated Results from the Phase 2/3 ALD-102 Study and First Report on Safety Outcomes from the Phase 3 ALD-104 Study

2021
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Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy

2020 Biology of Blood and Marrow Transplantation
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Haematopoietic stem cell transplantation in CSF1R-related adult-onset leukoencephalopathy with axonal spheroids and pigmented glia

2019 Journal of Neurology, Neurosurgery, and Psychiatry
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Giant axonal neuropathy: a multicenter retrospective study with genotypic spectrum expansion

2019 Neurogenetics
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Survival and Functional Outcomes in Boys with Cerebral Adrenoleukodystrophy with and without Hematopoietic Stem Cell Transplantation

2019 Biology of Blood and Marrow Transplantation
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Mutation ephb4 responsable de malformation anévrysmale de la veine de Galien

2019 Journal of Neuroradiology
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Microcephaly Modeling of Kinetochore Mutation Reveals a Brain-Specific Phenotype

2018 Cell Reports
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Loss of function mutations in EPHB4 are responsible for vein of Galen aneurysmal malformation.

2018 Brain
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Intracerebral gene therapy in children with metachromatic leukodystrophy: Results of a phase I/II trial

2018 Molecular Genetics and Metabolism
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