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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
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    • 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

Design of a polyepitope construct for the induction of HLA-A0201-restricted HIV 1-specific CTL responses using HLA-A*0201 transgenic, H-2 class I KO mice

February 19, 2021

Trafficking and propagation of canine adenovirus vectors lacking a known integrin-interacting motif

February 19, 2021

Trafficking and propagation of canine adenovirus vectors lacking a known integrin-interacting motif

February 19, 2021

Fetal somatic gene therapy – A preventive approach to the treatment of genetic disease

February 19, 2021

Transfert de gène au moyen de vecteurs adéno-associés

February 19, 2021

Intracellular trafficking of adeno-associated virus vectors: Routing to the late endosomal compartment and proteasome degradation

February 19, 2021

Mutations in the fukutin-related protein gene (FKRP) identify limb girdle muscular dystrophy 2I as a milder allelic variant of congenital muscular dystrophy MDC1C

February 19, 2021

Human cytomegalovirus protein US2 interferes with the expression of human HFE, a nonclassical class I major histocompatibility complex molecule that regulates iron homeostasis

February 19, 2021

Evidence for a digenic inheritance in a family with both febrile convulsions and temporal lobe epilepsy implicating chromosomes 18qter and 1q25-q31

February 19, 2021

First genetic evidence of GABAa receptor dysfunction in epilepsy: A mutation in the gamma2-subunit gene

February 19, 2021

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