Treatment of GM2 Gangliosidosis in Adult Sandhoff Mice Using an Intravenous Self-Complementary Hexosaminidase Vector

Karlaina J.L. Osmon, Patrick Thompson, Evan Woodley, Subha Karumuthil-Melethil, Cliff Heindel, John G. Keimel, William F. Kaemmerer, Steven J. Gray, Jagdeep S. Walia

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