authors

• Weiss, Lan
• Fazeli, Yasamin
• Chang, Mindy
• Paguio, Marvin
• Lee, Jonathan
• Yu, Howard
• Weiss, John
• Grossman, Tamar R
• Raben, Nina
• Jafar-Nejad, Paymaan
• Kimonis, Virginia
• Carrer, Michele
• Shmara, Alyaa
• Martin, Angela
• Yin, Hong
• Pal, Pallabi
• Cheng, Cheng
• Ta, Lac
• Boock, Victoria

publication date

• April 2025

published in

• Clinical and translational medicine  Journal

has subject area

• Animals
• Disease Models, Animal
• Glycogen
• Glycogen Storage Disease Type II
• Glycogen Synthase
• Humans
• Mice
• Mice, Knockout
• Muscle, Skeletal
• Oligonucleotides, Antisense
• alpha-Glucosidases

Keywords

• Enzyme replacement therapy (ERT)
• Gaa‐/‐ mouse model
• Pompe disease
• antisense oligonucleotides (ASOs)
• glycogen synthase 1 (GYS1)
• skeletal muscle

Digital Object Identifier (DOI)

• https://doi.org/10.1002/ctm2.70314

PubMed ID

• 40268518

start page

end page

volume

• 15

issue

• 4