Article
Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates.
Human gene therapy - 1 Jan 2016
Borel Florie, Gernoux Gwladys, Cardozo Brynn, Metterville Jake P, Toro Cabrera Gabriela C, Song Lina, Su Qin, Gao Guang Ping, Elmallah Mai K, Brown Robert H, Mueller Christian
Abstract excerpt
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease; survival in ALS is typically 3-5 years. No treatment extends patient survival by more than three months. Approximately 20% of familial ALS and 1-3% of sporadic ALS patients carry a mutation in the gene encoding superoxide dismutase 1 (SOD1). In a transgenic ALS mouse model expressing the mutant SOD1(G93A) protein, silencing the SOD1 gene...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.