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Archives: Publication

Assessment of Therapeutic Potential of a Dual AAV Approach for Duchenne Muscular Dystrophy

September 7, 2023

Liver-Directed Adeno-Associated Virus–Mediated
Gene Therapy for Mucopolysaccharidosis Type VI

May 3, 2023

Generation of two isogenic induced pluripotent stem cell lines from a 1-month-old nemaline myopathy patient harbouring a homozygous recessive c.121C > T (p.Arg39Ter) variant in the ACTA1 gene

May 3, 2023

Artificial intelligence workflow quantifying muscle features on Hematoxylin-Eosin stained sections reveals dystrophic phenotype amelioration upon treatment

May 3, 2023

Global genome decompaction leads to stochastic activation of gene expression as a first step toward fate commitment in human hematopoietic cells

May 3, 2023

Generation of an induced pluripotent stem cell line from a 3-month-old nemaline myopathy patient with a heterozygous dominant c.515C > A (p.Ala172Glu) variant in the ACTA1 gene

May 3, 2023

Hematopoietic stem and progenitors cells gene editing: Beyond blood disorders

May 3, 2023

What’s next in gene therapy for Crigler-Najjar syndrome?

May 3, 2023

Disrupted T-tubular network accounts for asynchronous calcium release in MTM1-deficient skeletal muscle

April 19, 2023

LSD1/PRMT6-targeting gene therapy to attenuate androgen receptor toxic gain-of-function ameliorates spinobulbar muscular atrophy phenotypes in flies and mice

April 19, 2023

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Who are we?

Our history Board of Directors The Management committee and scientific leadership Our scientific advisory board

Our science

Our research teams and our platforms Bioproduction Understanding gene therapy Gene Editing 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 Chronic septic granulomatosis Cori Forbes disease Crigler-Najjar syndrome Pompe disease Leber Hereditary Optic Neuropathy

Our commitment

Access to treatments Societal commitment Informing and sharing

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