Characterization of Immune Responses to rAAVrh8 Gene Therapy for GM2 Gangliosidosis in Phase 1/2 Trial
Terence R. Flotte, Meghan Blackwood, Motahareh Arjomandnejad, Ashley L. Harkins, Katelyn Sylvia, Sukanya Iyer, Danielle Kokoski, Rebecca Artinian, Allison M. Keeler
This study characterizes immune responses to AAVrh8 gene therapy in CNS patients and evaluates the effectiveness of immunosuppression.
medRxiv · May 7, 2025 · PreprintGenetic & Genomic Medicine