AAV10-U7-hSOD1

AAV10-U7-hSOD1 is an experimental gene therapy designed to treat familial amyotrophic lateral sclerosis (ALS) linked to mutations in the superoxide dismutase 1 (SOD1) gene. The therapy uses an adeno-associated virus serotype rh10 (AAVrh10, also referred to as AAV10) vector to deliver a modified U7 small nuclear RNA (snRNA) carrying antisense sequences targeting exon 2 of the human SOD1 pre-mRNA. This approach induces exon skipping, resulting in a premature stop codon and subsequent degradation of the mutant SOD1 transcript via nonsense-mediated decay. The reduction of toxic mutant SOD1 protein levels leads to improved neuromuscular function and prolonged survival in ALS mouse models[1][2][4]. This strategy represents a novel application of U7 snRNA-based splicing modulation for neurodegenerative disease.