GYS1 antisense oligonucleotide

GYS1 antisense oligonucleotides are gene-targeting therapeutics designed to selectively downregulate the expression of glycogen synthase 1 (GYS1), the primary enzyme responsible for glycogen synthesis in skeletal muscle and the brain. By binding to GYS1 mRNA and inducing RNase H1-mediated degradation, these antisense oligonucleotides (ASOs) act as substrate reduction therapies (SRT) to prevent the toxic accumulation of glycogen or abnormal polyglucosans. This therapeutic approach is being developed by Ionis Pharmaceuticals and Elpida Therapeutics, with the lead clinical candidate ION283 currently in Phase 1/2 development for Lafora disease (a fatal progressive myoclonus epilepsy characterized by neurotoxic Lafora bodies). Additionally, GYS1 ASOs are being investigated preclinically for Pompe disease as an adjunct to enzyme replacement therapy (ERT) to address persistent skeletal muscle pathology, as well as other glycogen storage disorders like glycogen storage disease type IV (GSD IV) and adult polyglucosan body disease (APBD).

Other names

glycogen synthase 1 antisense oligonucleotideGys1-ASOGys-1-ASOGys 1-ASOGYS1 ASOGYS-1 ASOGYS 1 ASO