Safety, tolerability and biological activity of ABX1100, a CD71 centyrin siRNA conjugate targeting GYS1 in late-onset Pompe disease patients

Molecular Genetics and Metabolism 2026

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Authors: Özlem Göker-Alpan, Tahseen Mozaffar, Melissa Brookes, Susan E. Sparks, Karyn T. O’Neil, Alan Pestronk, Aneal Khan

DOI: 10.1016/j.ymgme.2025.109435

Cited by: 0

Fields of Study & Subjects

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