Investigating Immune Responses to the scAAV9-<i>HEXM</i> Gene Therapy Treatment in Tay–Sachs Disease and Sandhoff Disease Mouse Models
GM2 gangliosidosis disorders are a group of neurodegenerative diseases that result from a functional deficiency of the enzyme β-hexosaminidase A (HexA). HexA consists of an α- and β-subunit; a deficiency in either subunit results in Tay–Sachs Disease (TSD) or Sandhoff Disease (SD), respectively. Vir...
Main Authors: | Shalini Kot, Subha Karumuthil-Melethil, Evan Woodley, Violeta Zaric, Patrick Thompson, Zhilin Chen, Erik Lykken, John G. Keimel, William F. Kaemmerer, Steven J. Gray, Jagdeep S. Walia |
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Format: | Article |
Language: | English |
Published: |
MDPI AG
2021-06-01
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Series: | International Journal of Molecular Sciences |
Subjects: | |
Online Access: | https://www.mdpi.com/1422-0067/22/13/6751 |
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