Pharmacological chaperones for human α-N-acetylgalactosaminidase

Schindler/Kanzaki disease is an inherited metabolic disease with no current treatment options. This neurologic disease results from a defect in the lysosomal α-N-acetylgalactosaminidase (α-NAGAL) enzyme. In this report, we show evidence that the iminosugar DGJNAc can inhibit, stabilize, and chaperon...

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Main Authors: Clark, Nathaniel E., Metcalf, Matthew C., Best, Daniel, Fleet, George W. J., Garman, Scott C.
Format: Artigo
Jezik:Inglês
Izdano: National Academy of Sciences 2012
Teme:
Biological Sciences
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC3491485/
https://ncbi.nlm.nih.gov/pubmed/23045655
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1203924109
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