P.91Optimization of AAV-mediated gene therapy for SOD1-linked ALS

Amyotrophic Lateral Sclerosis (ALS) is an incurable motor neuron (MN) disorder, characterized by degeneration of MNs leading to progressive paralysis and death within two to five years after diagnosis. Gene therapy is emerging as promising treatment option for patients affected by familial forms of ALS (fALS), representing 10% of all ALS cases. Taking advantage of viral vectors derived from Adeno-Associated Virus serotype 10 (AAV10) and the small nuclear RNA U7, we developed a gene therapy for SOD1-linked ALS (20% of fALS).

Source: Neuromuscular Disorders - September 30, 2019 Category: Neurology Authors: M. Biferi, M. Cohen-Tannoudji, T. Marais, B. Giroux, S. Astord Source Type: research