Investigating Immune Responses to the scAAV9-HEXM 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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Veröffentlicht in:Int J Mol Sci
Hauptverfasser: Kot, Shalini, Karumuthil-Melethil, Subha, Woodley, Evan, Zaric, Violeta, Thompson, Patrick, Chen, Zhilin, Lykken, Erik, Keimel, John G., Kaemmerer, William F., Gray, Steven J., Walia, Jagdeep S.
Format: Artigo
Sprache:Inglês
Veröffentlicht: MDPI 2021
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC8268035/
https://ncbi.nlm.nih.gov/pubmed/34201771
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms22136751
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