Galactosemia: Towards Pharmacological Chaperones
نویسندگان
چکیده
منابع مشابه
Pharmacological Chaperones by Design
One of the most successful approaches in the synthesis of inhibitors of t he g lycosidases i s t he s ubstitution of t he e ndocyclic ox ygen i n monosaccharides by a nitrogen atom to get iminosugars. Given that many diseases h ave t heir o rigin i n t he malfunctioning of t hese e nzymes, t his glycomimetics bear s trong p otential a s dr ug c andidates. A t th eir p rotonated state, iminosuga...
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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 (currentl...
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Most Pharmacological chaperones (PC’s) described until now are substrate analogues which bind to the active site of the target protein. Consequently, such PC’s also inhibit the target protein at higher concentrations thus rendering a narrow therapeutic window and have poor drug-like properties. Through our proprietary technology platform SEE-TxTM, we identify a new generation of non-substrate c...
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ژورنال
عنوان ژورنال: Journal of Personalized Medicine
سال: 2021
ISSN: 2075-4426
DOI: 10.3390/jpm11020106