Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease

Abstract Pompe disease (PD) is a progressive myopathy caused by the aberrant accumulation of glycogen in skeletal and cardiac muscle resulting from the deficiency of the enzyme acid alpha‐glucosidase (GAA). Administration of recombinant human GAA as enzyme replacement therapy (ERT) works well in all...

Full description

Saved in:

Bibliographic Details
Main Authors:	Lan Weiss, Michele Carrer, Alyaa Shmara, Angela Martin, Hong Yin, Pallabi Pal, Cheng Cheng, Lac Ta, Victoria Boock, Yasamin Fazeli, Mindy Chang, Marvin Paguio, Jonathan Lee, Howard Yu, John Weiss, Tamar R Grossman, Nina Raben, Paymaan Jafar‐Nejad, Virginia Kimonis
Format:	Article
Language:	English
Published:	Wiley 2025-04-01
Series:	Clinical and Translational Medicine
Subjects:	antisense oligonucleotides (ASOs) Enzyme replacement therapy (ERT) Gaa‐/‐ mouse model glycogen synthase 1 (GYS1) Pompe disease skeletal muscle
Online Access:	https://doi.org/10.1002/ctm2.70314
Tags:	Add Tag No Tags, Be the first to tag this record!

Be the first to leave a comment!

Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease

Similar Items