AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial
Connor J. Lewis, Precilla D’Souza, Jean M. Johnston, Maria T. Acosta, Cristan Farmer, Eva H. Baker, Anna Crowell, Yoliann Mojica Algarin, Sumaiya Ashraf, Lisa A. Joseph, Gilbert Vézina, Zenaide Quezado, Muhammad H. Yousef, Zeynep Vardar, Mohammed Salman Shazeeb, , Cynthia J. Tifft
This phase 1/2 trial evaluates AAV9 gene therapy for GM1 gangliosidosis Type II, showing safety and some biochemical and neuroimaging improvements.
New England Journal of Medicine · Feb 6, 2026Peer-reviewedGenetic & Genomic Medicine