Treating ALS by gene therapy : a true hope
Amyotrophic lateral sclerosis (ALS) affects approximately 1 in 25,000 people. This disease is caused by the loss of the motor neurons located in the spinal cord which lead to disabling symptoms, such as a muscle weakness and difficulties in walking or writing. The disease progresses between 2 and 5 years until the patient is paralyzed and dies from respiratory failure. Up to now, unfortunately, no therapy exists.
Our guest, Dr. Maria-Grazia Biferi, heads a research group at the Paris Institute of Myology, dedicated to the development of gene therapy for motor neuron diseases. The therapeutic method is based on the use of viral vectors : AAVs.
Discover the podcast (in French only):