Publication – Généthon

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