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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Detalhes bibliográficos

Principais autores:	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
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Wiley 2025-04-01
coleção:	Clinical and Translational Medicine
Assuntos:	antisense oligonucleotides (ASOs) Enzyme replacement therapy (ERT) Gaa‐/‐ mouse model glycogen synthase 1 (GYS1) Pompe disease skeletal muscle
Acesso em linha:	https://doi.org/10.1002/ctm2.70314
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