Press Releases

AFM-Téléthon and the ‘Sociétés de Projets Industriels’ SPI [Industrial Projects Companies] investment fund, managed by Bpifrance under the Programme d’Investissement d’Avenir (PIA), are creating YposKesi, the first French company to develop and produce gene and cell therapy products. This new company aims to make the first treatments available to patients and market them at a … [Read more]

A study by Xavier Nissan’s research team from I-Stem, in collaboration with Pr Nicolas Lévy’s (Aix-Marseille University – INSERM UMR_S910, Faculté de Médecine de Marseille), Dr Lino Ferreira’s (Coimbra University, Portugal) teams enabled to show the therapeutic potential of retinoic acid (Vitamin A) in the treatment of progeria. Hutchinson-Gilford progeria syndrome is a rare genetic … [Read more]

Dr. Federico MINGOZZI, Team Leader of the Immunology and Liver Gene Transfer unit at Généthon, (Inserm U951/UPMC), France, has demonstrated the long-term efficacy of an optimized AAV-UGT1A1 vector for the correction of Crigler-Najjar syndrome (CN) in two different animal models of the disease. This work, published in July 20, 2016 in Molecular Therapy Methods and … [Read more]

A team from the Institute of Myology research center called “RNA-repair based therapeutic strategies and skeletal muscle pathophysiology, in a study led and conducted by Stéphanie Lorain and Cécile Peccate (UPMC / Inserm / CNRS / Institute of Myology), showed that a pre-treatment made of antisense oligonucleotides improves significantly the benefit of gene therapy for … [Read more]

A team from the Institute of Myology, leaded by France Piétri-Rouxel (Sorbonne Universities, Pierre and Marie Curie University / Inserm / CNRS / Institute of Myology) enabled to define a threshold of muscular fibres containing the dystrophin needed to normalize the condition of proteins essential to the contractile function of muscles, but also to significantly … [Read more]

In I-Stem, a team led by Alexandra Benchoua and Marc Pechanski enabled to prove the benefit of neuronal and celullar models derived from pluripotent stem cells for genetic autistic disorders. These researches were focused on the Phelan-McDernid, a rare autistic disease of genetic origins which leads to a loss of one of the copies of … [Read more]

A research team from the Institute of Myology, led by Denis Furling, has taken part in a collaborative work coordinated by Nicolas Charlet (Institute of Genetics and Molecular and Cellular Biology, Strasbourg) and Masanori Takahashi (Osaka, Japan), which enabled to bring to light the key role of alteration (with expanded CTG repeats) of the splicing … [Read more]