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DMD: efficacy of gene augmentation therapy through a pre-treatment with antisense oligonucleotides

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]

Determining of the level of dystrophin essential to restore a physiological condition needed to the contractile function of muscles in GRMD dogs

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]

An I-Stem team opens the way towards high-precision medicine

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]

AIRE, key factor in the gender inequality for autoimmune diseases

Nadine Dragin, researcher among an Inserm/UPMC/CNRS (French National Center for Scientific Research)/Institute of Myology team co-directed by Sonia Berrih-Aknin and Rozen le Panse from the Institute of Myology, based in the Pitié-Salpêtrière Hospital, AP-HP, brought to light the central role of AIRE, a key factor for immune tolerance, in the gender inequality regarding autoimmune diseases. … [Read more]

Alteration of SCN5A splicing causing prevailing heart disorders in Myotonic Dystrophy

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]

Postdoctoral Programs

Spring 2016 The Genethon International Postdoctoral Program 2016 is now open. Positions are intended for highly motivated scientists who wish to pursue their career in the research and development of therapies for rare diseases. Postdoctoral research positions are available for a period of three years (37-41 k€ annual gross income depending on qualifications and years … [Read more]

Focus Researcher: Charles Gersbach at Myology 2016

Charles Gersbach on the use of gene correction and gene editing technologies to correct mutations in genetic diseases.

Myology 2016 – Day 5: Focus on the heart and advanced therapies

The congress Myology 2016, organised by AFM-Téléthon, is now coming to an end: over 900 international experts in the field (researchers, clinicians, doctors…) have compared their latest work for 5 days. This edition was undoubtedly marked by the beginning of the therapeutic era for neuromuscular diseases with the presentation of clinical trials results in human … [Read more]

Myology 2016 – Day 4: Focus on therapeutics on trial

The fourth day of Myology 2016, organised by AFM-Téléthon, opened on a plenary session dedicated to the different myology-related advances. H. Lee Sweeney from the University of Florida (USA), co-chairman of the congress, overviewed the trials using oligonucleotides in Duchenne muscular dystrophy. He underlined the need, for demonstrating the efficiency of products currently under clinical … [Read more]

Focus Researcher: Jerry Mendell at Myology 2016

Jerry Mendell told us about gene therapy in spinal muscular atrophy.