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Target intelligence / Profile preview
Hexosaminidase Subunit Alpha (HEXA)
- Target
- HEXA
- Molecular classification
- Enzyme, Glycosyl hydrolase
01
Overview
The HEXA gene encodes the alpha subunit of beta-hexosaminidase A, a lysosomal enzyme that is essential for breaking down GM2 ganglioside. Mutations in HEXA lead to Tay-Sachs disease, characterized by the accumulation of GM2 ganglioside in neurons, causing neurodegeneration.
- Other names
- TSDhexosaminidase subunit alpha
02
Mechanism of action
N/A
03
Biological functions
Lysosomal degradationGM2 ganglioside catabolismNeuronal health maintenance
04
Disease associations
Tay-Sachs DiseaseGM2-Gangliosidosis
05
Safety considerations
Off-target effects of therapiesDelivery of therapies to the central nervous systemSpecificity of therapies for different mutations
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