Molecular therapy : the journal of the American Society of Gene Therapy
AAV gene therapy for GBA1-related diseases.
Swathi Ayloo, Jae Cheon Ryu, Shih-Ching Chou, Lydia Blatnik, Erik Wischhof, Jie Bu, Lilu Guo, Mahmud Hossain, Dhiman Ghosh, William McCarty, Ann Byrne, Lilly Chai, Jennifer Clarke, Can Kayatekin, Bailin Zhang
Published: 202510.1016/j.ymthe.2025.12.011
Abstract
Mutations in GBA1, the gene encoding glucocerebrosidase (GCase), are the most common risk factor for Parkinson's disease (PD). GBA-PD patients are a genetic subpopulation of PD carrying heterozygous mutations in GBA1. Additionally, bi-allelic mutatio…
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