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      • Progressive muscular dystrophies
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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
    • Sickle cell disease
    • Chronic septic granulomatosis
    • Severe Combined Immunodefiency (SCID)
    • Cori Forbes disease
    • GSD1a – Glygogen Storage Disease Type 1a
    • Crigler-Najjar syndrome
    • Pompe disease
    • Leber Hereditary Optic Neuropathy
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Archives: Publication

Targeting Mitochondrial Defects to Increase Longevity in Animal Models of Neurodegenerative Diseases.

February 19, 2021

Dual muscle-liver transduction imposes immune tolerance for muscle transgene engraftment despite preexisting immunity.

February 19, 2021

Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome.

February 19, 2021

Towards a scalable purification protocol of GaLV-TR pseudotyped lentiviral vectors.

February 19, 2021

Breaking the Barriers of Genetic and Metabolic Disorders.

February 19, 2021

X-linked myotubular myopathy: A prospective international natural history study.

February 19, 2021

234th ENMC International Workshop: Chaperone dysfunction in muscle disease Naarden, The Netherlands, 8-10 December 2017.

February 19, 2021

Liver-directed gene therapy results in long-term correction of progressive familial intrahepatic cholestasis type 3 in mice.

February 19, 2021

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype.

February 19, 2021

Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study.

February 19, 2021

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