Genetic & Genomic MedicinemedRxivPreprint
Characterization of Immune Responses to rAAVrh8 Gene Therapy for GM2 Gangliosidosis in Phase 1/2 Trial
Terence R. Flotte, Meghan Blackwood, Motahareh Arjomandnejad…·Allison M. Keeler
This study characterizes immune responses to AAVrh8 gene therapy in CNS patients and evaluates the effectiveness of immunosuppression.
2 citations