Three classes of glucocerebrosidase inhibitors identified by quantitative high-throughput screening are chaperone leads for Gaucher disease

Gaucher disease is an autosomal recessive lysosomal storage disorder caused by mutations in the glucocerebrosidase gene. Missense mutations result in reduced enzyme activity that may be due to misfolding, raising the possibility of small-molecule chaperone correction of the defect. Screening large c...

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Bibliographische Detailangaben
Hauptverfasser: Zheng, Wei, Padia, Janak, Urban, Daniel J., Jadhav, Ajit, Goker-Alpan, Ozlem, Simeonov, Anton, Goldin, Ehud, Auld, Douglas, LaMarca, Mary E., Inglese, James, Austin, Christopher P., Sidransky, Ellen
Format: Artigo
Sprache:Inglês
Veröffentlicht: National Academy of Sciences 2007
Schlagworte:
Biological Sciences
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC1936979/
https://ncbi.nlm.nih.gov/pubmed/17670938
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0705637104
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