AAV9-siRNA-Gys1

AAV9-siRNA-Gys1 is an experimental adeno-associated virus serotype 9 (AAV9) gene therapy designed as a substrate reduction therapy (SRT) for Pompe disease. Developed by researchers at the University of Texas Southwestern Medical Center, the therapy intravenously delivers a small interfering RNA (siRNA) targeting glycogen synthase 1 (Gys1), which is the rate-limiting enzyme in glycogen synthesis. By downregulating Gys1, AAV9-siRNA-Gys1 reduces the synthesis of glycogen, thereby limiting its pathogenic accumulation in the lysosomes of skeletal and cardiac muscles. In preclinical studies using neonatal Pompe disease mouse models, the therapy demonstrated significant histological, biochemical, and functional improvements, suggesting its potential as a complementary approach to standard enzyme replacement therapy (ERT).

Other names

AAV9-mediated Gys1 siRNAAAV-9-mediated Gys1 siRNAAAV 9-mediated Gys1 siRNAAAV9-mediated RNAi targeting of Gys1AAV-9-mediated RNAi targeting of Gys1AAV 9-mediated RNAi targeting of Gys1