Novel Patient Cell-Based HTS Assay for Identification of Small Molecules for a Lysosomal Storage Disease

Small molecules have been identified as potential therapeutic agents for lysosomal storage diseases (LSDs), inherited metabolic disorders caused by defects in proteins that result in lysosome dysfunctional. Some small molecules function assisting the folding of mutant misfolded lysosomal enzymes tha...

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Detaylı Bibliyografya
Asıl Yazarlar: Geng, Haifeng, Whiteley, Grace, Ribbens, Jameson, Zheng, Wei, Southall, Noel, Hu, Xin, Marugan, Juan J., Ferrer, Marc, Maegawa, Gustavo H. B.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Public Library of Science 2011
Konular:
Research Article
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC3244463/
https://ncbi.nlm.nih.gov/pubmed/22216298
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0029504
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