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    • Gene Editing
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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

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

Dystrophin myonuclear domain restoration governs treatment efficacy in dystrophic muscle

April 19, 2023

Gene therapy review: Duchenne muscular dystrophy case study

April 19, 2023

Dlk1-Dio3 cluster miRNAs regulate mitochondrial functions in the dystrophic muscle in Duchenne muscular dystrophy

February 22, 2023

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