Dual-vector rAAVrh8 gene therapy for GM2 Gangliosidosis: a Phase I/II trial
Terence R. Flotte, MD
This is a dose escalation of a dual AAV vector gene therapy for Tay-Sachs and Sandhoff diseases in which a novel bithalamic infusion process was used. The broad distribution of axonal connections of thalamic neurons enables transport of the normal B-hexosaminidase A enzyme throughout the cerebrum.