SODr146192

SODr146192 is a rodent-specific antisense oligonucleotide (ASO) designed to target and reduce the expression of superoxide dismutase 1 (SOD1). It is a 20-nucleotide phosphorothioate-modified oligonucleotide featuring 2'-O-methoxyethyl (MOE) gapmer technology, which facilitates the RNase H-mediated degradation of SOD1 mRNA. Developed by Ionis Pharmaceuticals (formerly Isis Pharmaceuticals) in collaboration with researchers at the University of California San Diego, SODr146192 was utilized in pivotal preclinical studies to demonstrate that the targeted reduction of SOD1 levels within the central nervous system could significantly slow disease progression and extend survival in mouse models of amyotrophic lateral sclerosis (ALS). The compound is typically administered via continuous intraventricular or intrathecal infusion to ensure broad distribution throughout the brain and spinal cord. While SODr146192 serves as a research tool for rodent models, it provided the critical proof-of-concept for the subsequent development of human-targeted SOD1 ASOs, such as tofersen (BIIB067).

Other names

SOD1 ASOSOD-1 ASOSOD 1 ASOrodent SOD1 antisense oligonucleotide