Article

Human Recombinant Lysosomal β-Hexosaminidase A produced in P.pastoris efficiently reduced GM2 ganglioside accumulation in Tay- Sachs Disease

2024-07-12

Abstract excerpt

<title>Abstract</title> <p>Tay-Sachs disease is a progressive lysosomal storage disorder caused by genetic mutations in the HEXA gene encoding α-subunit of β-Hexosaminidase A, which leads to GM2 ganglioside accumulation, particularly in the central nervous system. Lysosomal GM2 ganglioside accumulation causes neuropathology and leads to premature cell death in Tay-Sachs patients, and there is no effective treatme...

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Literature Corpus work: 1a481033-9926-5bfb-a076-c775f73399cc
DOI: 10.21203/rs.3.rs-4577970/v1

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