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

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...

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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
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https://doi.org/10.1002/ctm2.70314

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