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    • Duchenne muscular dystrophy
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    • Myotubular myopathy
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    • Cori Forbes disease
    • GSD1a – Glygogen Storage Disease Type 1a
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Archives: Publication

Severe hematopoeitic stem cell inflammation compromises chronic granulomatous disease gene therapy

February 22, 2023

Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome

February 22, 2023

Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques

December 20, 2022

Objective evaluation of clinical actionability for genes involved in myopathies: 63 genes with a medical value for patient care

December 20, 2022

Mapping genomic loci implicates genes and synaptic biology in schizophrenia

December 20, 2022

The Amyotrophic Lateral Sclerosis M114T PFN1 Mutation Deregulates Alternative Autophagy Pathways and Mitochondrial Homeostasis

December 20, 2022

Rescue of a familial dysautonomia mouse model by AAV9-Exon-specific U1 snRNA

December 20, 2022

Le registre national des calpaïnopathies 

December 20, 2022

Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease

December 20, 2022

Severe ACTA1-related nemaline myopathy: intranuclear rods, cytoplasmic bodies, and enlarged perinuclear space as characteristic pathological features on muscle biopsies

December 20, 2022

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