Gys1 ASO#1

Gys1 ASO#1 is an antisense oligonucleotide (ASO) designed to target and knock down the expression of glycogen synthase 1 (GYS1), the enzyme responsible for glycogen synthesis in skeletal muscle. It is being investigated as a substrate reduction therapy (SRT) for Pompe disease, a lysosomal storage disorder caused by acid alpha-glucosidase (GAA) deficiency. By reducing GYS1 levels, the drug aims to curb the aberrant accumulation of glycogen in skeletal muscle, which is often poorly addressed by standard enzyme replacement therapy (ERT). In preclinical studies using Gaa-/- mouse models, Gys1 ASO#1 has demonstrated the ability to reduce muscle glycogen content, alleviate autophagic buildup, and improve motor function, especially when used in combination with ERT. The ASO is designed to be specific for GYS1 to avoid interfering with the liver-specific isoform, GYS2.

Other names

GYS1-targeting antisense oligonucleotideGYS-1-targeting antisense oligonucleotideGYS 1-targeting antisense oligonucleotide