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Journée internationale Maladies Rares du 28 février 2010 : Les acteurs de la Plateforme Maladies Rares attendent un second plan français à la hauteur de enjeux

Read the press release – 18/02/10 (1 page – 39 ko) [FR]

An effective gene therapy approach for dysferlin deficiencies

Research scientists at Généthon find a solution to the problem of the dysferlin gene’s size to transport it into muscle A team of researchers led by Isabelle Richard (CNRS UMR8587 LAMBE) in the Généthon laboratory, created and funded by AFM through Téléthon, has just demonstrated the efficacy in animals of a gene therapy strategy for … [Read more]

Thermo Fischer Scientific 2009 prize

Anne Galy, winner of the Biotherapy prize for her project “gene therapy in Wiskott-Aldrich syndrome” On 22 June, at the 8th annual conference of the “Société Francophone de Thérapie Cellulaire et Génique” (French-speaking Cellular and Gene Therapy Society) in Paris, Anne Galy, Généthon research director of the Inserm unit, will be presented the 2009 Biotherapy … [Read more]

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]