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: | , , , , |
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| Format: | Journal article |
| Language: | English |
| Published: |
2012
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