Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease

Subha Karumuthil-Melethil, Sahana Nagabhushan Kalburgi, Patrick Thompson, Michael Tropak, Michael D. Kaytor, John G. Keimel, Brian L. Mark, Don Mahuran, Jagdeep S. Walia, Steven J. Gray