Investigating Immune Responses to the scAAV9-<i>HEXM</i> Gene Therapy Treatment in Tay–Sachs Disease and Sandhoff Disease Mouse Models

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...

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Bibliographic Details
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
Format: Article
Language:English
Published: MDPI AG 2021-06-01
Series:International Journal of Molecular Sciences
Subjects:
GM2
gangliosidosis
Tay-Sachs
Sandhoff
scAAV9-<i>HEXM</i>
HexM
Online Access:https://www.mdpi.com/1422-0067/22/13/6751

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https://www.mdpi.com/1422-0067/22/13/6751

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