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Lessons learned from clinical experience with AAV vectors.

2023

Gardin A et AL.

Archives de pediatrie : organe officiel de la Societe francaise de pediatrie 30, 8S46-48S52.

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Our pipeline

Duchenne muscular dystrophy Infant Spinal muscular atrophy SMA-PME and Farber’s disease Myotubular myopathy Limb girdle muscular dystrophies Wiskott-Aldrich syndrome Fanconi’s anemia Chronic septic granulomatosis Cori Forbes disease Crigler-Najjar syndrome Pompe disease Leber Hereditary Optic Neuropathy

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Genethon is a member of the Biotherapies Institute for Rare Diseases that was created by AFM-Telethon

Biotherapies Institute

Institute of Myology

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