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    • Our history
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    • Our research teams and our platforms
      • Progressive muscular dystrophies
      • Neuromuscular diseases and gene therapy
      • Gene Editing
      • Immunology and liver diseases
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    • Gene Editing
    • Bioproduction
    • Understanding gene therapy
    • Immunology of gene transfer: a crucial issue
  • 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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    • Access to treatments
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Archives: Publication

Deciphering the Molecular Mechanism of Incurable Muscle Disease by a Novel Method for the Interpretation of miRNA Dysregulation

December 20, 2022

Dual Blockade of Misfolded Alpha-Sarcoglycan Degradation by Bortezomib and Givinostat Combination

December 20, 2022

Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients

December 20, 2022

Cytokines, chemokines and growth factors profile in human aqueous humor in idiopathic uveitis

December 20, 2022

Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders

December 20, 2022

Lentiviral standards to determine the sensitivity of assays that quantify lentiviral vector copy numbers and genomic insertion sites in cells

December 20, 2022

Skeletal Muscle Cells Derived from Induced Pluripotent Stem Cells: A Platform for Limb Girdle Muscular Dystrophies

December 20, 2022

Co-Administration of Simvastatin Does Not Potentiate the Benefit of Gene Therapy in the mdx Mouse Model for Duchenne Muscular Dystrophy

December 20, 2022

Base-editing-mediated dissection of a gamma-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression

December 20, 2022

CRISPRthripsis: “The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy

December 20, 2022

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