Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease
Lan Nguyen Weiss, Michele Carrer, Alyaa Shmara, Angela Martin, Hong Zhen Yin, Pallabi Pal, Cheng Cheng, Lac Ta, Victoria Boock, Yasamin Fazeli, Mindy Chang, Marvin Paguio, Jonathan Lee, Howard Yu, John H. Weiss, , Virginia E. Kimonis
This study shows that ASO-mediated Gys1 knockdown in a mouse model of Pompe disease reduces glycogen accumulation and improves motor function, both alone and in combination with ERT.
Clinical and Translational Medicine · Apr 1, 2025Peer-reviewedMolecular Biology