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Limb-Girdle Muscular Dystrophy Type 2I/R9: first gene therapy results

The first clinical results of the gene therapy for the treatment of limb-girdle muscular dystrophy type 2I/R9 show good preliminary efficacy and biological tolerability. These first results have been presented during oral communications during the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) and will be presented in the 2023 … [Read more]

Human induced pluripotent stem cells, a valuable tool for better understanding and treating neuromuscular diseases

A review published by two researchers at I-Stem, one of the AFM-Téléthon laboratories, takes stock of human pluripotent stem cells (hiPS) and confirms their main advantages in neuromuscular diseases: improving understanding and identifying new treatments. Read the news

Crigler-Najjar: gene therapy shows its effectiveness

« Immunologie et Thérapie Génique des Maladies du Foie » de Généthon, dirigée par le Dr Giuseppe Ronzitti Results published in The New England Journal of Medicine demonstrate the safety and tolerance for gene therapy in Crigler-Najjar syndrome as well as its efficacy at the highest dose. This rare liver disease is characterized by a … [Read more]

A Genethon team has succeeded in inhibiting the immune response linked to AAV

A research team from Genethon, in collaboration with teams from CNRS/Inserm and from the biotechnology company Spark Therapeutics, announced in Nature Medicine that it has succeeded in inhibiting the immune response induced by AAV antibodies present as a result of natural immunity or following gene therapy, thanks to the IdeS enzyme. This result opens up … [Read more]

World premiere : differentiation of pluripotent stem cells to muscle fiber

This major breakthrough in neuromuscular diseases has just been published August 3, 2015 in Nature Biotechnology. “With our method, which does not require any genetic manipulation, we obtain muscle fibers which contract, but also satellite stem cells “ Olivier Pourquié, IGBMC and Harvard University (photo credit : Lola Vélasquez/IGBMC) More information with the original article  … [Read more]

A new trial in gene therapy led by UCLA in partnership with Genethon

10 children with an immunodeficiency disorder, commonly referred to as X-linked CGD, will be enrolled to receive a stem cell gene therapy, due to Dr Kohn’s team and Genethon. Genethon, AFM-Téléthon’s laboratory research, engineered the virus delivery system designed to transport the gene therapy into patients’ blood-forming stem cells. Genethon is sponsoring a parallel European … [Read more]

ASGCT 18 th Annual Meeting 2015: Ana Buj Bello receives the Outstanding New Investigator Award

This award recognizes four researchers every year for the quality and importance of their work in gene and cell therapy. Ana Buj Bello is awarded for 10 years of pioneering work on gene therapy and currently leads a translational gene therapy program for myotubular myopathy, a very severe muscular disease t due to mutations in … [Read more]