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...
Main Authors: | Clark, N, Metcalf, M, Best, D, Fleet, G, Garman, S |
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Format: | Journal article |
Language: | English |
Published: |
2012
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