Treating ALS by gene therapy : a reason for hope
Amyotrophic lateral sclerosis (ALS) affects approximately 1 in 25,000 people. This disease is caused by loss of motor neurons in the spinal cord, leading to disabling symptoms such as a muscle weakness and difficulties in walking or writing. ALS progresses between 2 and 5 years, until the patient becomes paralyzed and dies from respiratory failure. Unfortunately, at present, no cure exists.
Our podcast guest speaker is Dr. Maria-Grazia Biferi, who leads a research group at the Paris Institute of Myology dedicated to the development of gene therapy for motor neuron diseases. Their therapeutic method is based on the use of viral vectors : AAVs.
Listen to the podcast here (available in French only):