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      • Progressive muscular dystrophies
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    • Gene Editing
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    • 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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Archives: Publication

Germline duplication of ATG2B and GSKIP predisposes to familial myeloid malignancies.

February 19, 2021

A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence.

February 19, 2021

Lentiviral-Mediated Gene Therapy in Fanconi Anemia-A Mice Reveals Long-Term Engraftment and Continuous Turnover of Corrected HSCs.

February 19, 2021

The EuroBioBank Network: 10 years of hands-on experience of collaborative, transnational biobanking for rare diseases.

February 19, 2021

Detection of TRIM32 deletions in LGMD patients analyzed by a combined strategy of CGH array and massively parallel sequencing.

February 19, 2021

Genome-Wide Definition of Promoter and Enhancer Usage during Neural Induction of Human Embryonic Stem Cells.

February 19, 2021

Advances in cell culture: anchorage dependence.

February 19, 2021

Determination of anti-adeno-associated virus vector neutralizing antibody titer with an in vitro reporter system.

February 19, 2021

Adeno-Associated Viral Vectors at the Frontier between Tolerance and Immunity.

February 19, 2021

Adeno-associated virus vector-based gene therapy for monogenetic metabolic diseases of the liver.

February 19, 2021

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