Enhanced correction of skeletal muscle and brain pathology in a Pompe mouse model using transferrin receptor-mediated delivery of GAA

Molecular Genetics and Metabolism 2026

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Authors: Rashi Priya, Cinthia V. Pastuskovas, Hoang Nguyen, Isabel A. Becerra, Manuel Montalban, Rajarshi Ghosh, Chau Tran, Jamal I. Alkabsh, Sonnet S. Davis, Audrey G. Reeves, Romeo D. Maciuca, Tianao Yuan
+8 more Jung Hwa Suh, Meng Fang, Neal S. Gould, Anastasia G. Henry, Sarah Huntwork, Stephanie Coffin, Annie Arguello, Cathal S. Mahon
DOI: 10.1016/j.ymgme.2025.109588
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus RNA Interference and Gene Delivery

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109588–109588
Publication date
1 February 2026
Language
EN