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

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17 Scopus citations

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Medicine & Life Sciences