First advances in gene surgery

Duchenne muscular dystrophy (DMD) was the subject of the first gene surgery trials. It is a model disease for developing this innovative approach which will have much broader applications to diseases such as cancers, infectious diseases and heart disease.

Functional dystrophin restored by exon skipping

At the end of 2007, Prosensa and the University of Leiden (Netherlands) medical centre team published encouraging results of a first exon skipping trial of antisense oligonucleotides using intramuscular administration. Tolerance of the treatment was good and above all a truncated but functional form of the protein responsible for DMD (dystrophine) was produced in the four patients treated.

Another trial led by Prosensa showed that the same treatment administered subcutaneously allowed antisense oligonucleotides to be distributed throughout the body and dystrophin production was restored in the fifteen patients.

20 Mar 2018

The 31th Telethon edition reaches a final collection of 89,189,384 euros

On 8th and 9th December 2017, the Telethon enthralled the public throughout France.

14 Mar 2018

Spinal muscular atrophy : AveXis enters into licensing agreement with Genethon

AveXis, Inc. and Genethon today announced they have entered into an exclusive, worldwide license agreement for in vivo gene therapy delivery of AAV9 vector into the central nervous system (CNS) for the treatment of spinal muscular atrophy (SMA).

13 Feb 2018

Gene therapy: Généthon starts a clinical trial to test a gene therapy treatment for a rare liver disease, Crigler-Najjar Syndrome

Illustration for news: Gene therapy: Généthon starts a clinical trial to test a gene therapy treatment for a rare liver disease, Crigler-Najjar Syndrome

Généthon, a laboratory created by AFM-Téléthon, starts a European phase I/II clinical trial to test a treatment for Crigler-Najjar Syndrome, a rare liver disease.

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