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Gene therapy: Production of the first batch of HIV-derived vectors in Europe for use in a human clinical trial

Genethon, the laboratory created and funded by the AFM (French Association against Myopathies) using donations from Téléthon, today announced that it has produced, controlled and released a batch of lentiviral vectors derived from the human immunodeficiency (HIV) virus for a gene therapy trial in humans in a rare immune deficiency. The Etablissement de thérapie génique … [Read more]

Neuromuscular diseases: A pharmacological therapeutic approach for certain sarcoglycanopathies

A research team led by Dr Isabelle Richard (CNRS-FRE3087) from the Genethon laboratory financed by the AFM through Téléthon donations has just demonstrated the efficacy of a novel pharmacological strategy in the mouse for certain mutations of alpha-sarcoglycanopathy or LGMD2D, a recessive limb girdle dystrophy. This pharmacological strategy could be applied to other limb girdle … [Read more]