Preclinical evaluation of a durable, tissue-targeted gene therapy for Pompe disease demonstrates superior efficacy over standard ERT

Molecular Genetics and Metabolism 2026

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Authors: Maria Praggastis, Daniel M. Virga, Yuxin Zhu, Andrew D. Baik, Nyanza J. Rothman, Xiaojiao Xue, Katherine D. Cygnar

DOI: 10.1016/j.ymgme.2025.109587

Cited by: 0

Fields of Study & Subjects

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