Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice

Karlaina J.L. Osmon, Evan Woodley, Patrick Thompson, Katalina Ong, Subha Karumuthil-Melethil, John G. Keimel, Brian L. Mark, Don Mahuran, Steven J. Gray, Jagdeep S. Walia

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