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Article

Pharmacological chaperones for human α- <i>N</i> -acetylgalactosaminidase

8 Oct 2012

Abstract excerpt

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 chaperone human α-NAGAL both in vitro and in vivo. We demonstrate that a related iminosugar DGJ (currently in phase III...

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PMID: 23045655
DOI: 10.1073/pnas.1203924109

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