A new AAV10-mediated gene therapy for SOD1-linked ALS

Our research is devoted to the identification of efficient strategies to target the central nervous system (CNS) and to the development of novel therapies for motor neuron disorders. In particular, using the unique therapeutic potential of self-complementary adeno-associated virus (AAV) vectors, we recently elaborated a new gene therapy strategy for a genetic form of Amyotrophic Lateral Sclerosis (ALS), a lethal disease with limited therapeutic options. Approximately 20% of familial ALS cases are caused by mutations in the Superoxide Dismutase 1 (SOD1) gene and toxic gain of function of the mutant protein.

Source: Neuromuscular Disorders - September 10, 2017 Category: Neurology Authors: M. Biferi, M. Cohen-Tannoudji, A. Cappelletto, B. Giroux, M. Roda, S. Astord, T. Marais, A. Ferry, T. Voit, M. Barkats Source Type: research