piperidines and Tay-Sachs-Disease

piperidines has been researched along with Tay-Sachs-Disease* in 1 studies

Other Studies

1 other study(ies) available for piperidines and Tay-Sachs-Disease

Article	Year
sp Journal of enzyme inhibition and medicinal chemistry, 2022, Volume: 37, Issue:1 The late-onset form of Tay-Sachs disease displays when the activity levels of human β-hexosaminidase A (HexA) fall below 10% of normal, due to mutations that destabilise the native folded form of the enzyme and impair its trafficking to the lysosome. Competitive inhibitors of HexA can rescue disease-causative mutant HexA, bearing potential as pharmacological chaperones, but often also inhibit the enzyme O-glucosaminidase (GlcNAcase; OGA), a serious drawback for translation into the clinic. We have designed sp Topics: beta-N-Acetylhexosaminidases; Hexosaminidase A; Humans; Lysosomes; Piperidines; Tay-Sachs Disease	2022

Article

Year

Journal of enzyme inhibition and medicinal chemistry, 2022, Volume: 37, Issue:1

The late-onset form of Tay-Sachs disease displays when the activity levels of human β-hexosaminidase A (HexA) fall below 10% of normal, due to mutations that destabilise the native folded form of the enzyme and impair its trafficking to the lysosome. Competitive inhibitors of HexA can rescue disease-causative mutant HexA, bearing potential as pharmacological chaperones, but often also inhibit the enzyme O-glucosaminidase (GlcNAcase; OGA), a serious drawback for translation into the clinic. We have designed sp

Topics: beta-N-Acetylhexosaminidases; Hexosaminidase A; Humans; Lysosomes; Piperidines; Tay-Sachs Disease

2022