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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Detalles Bibliográficos
Autores principales: Geng, Haifeng, Whiteley, Grace, Ribbens, Jameson, Zheng, Wei, Southall, Noel, Hu, Xin, Marugan, Juan J., Ferrer, Marc, Maegawa, Gustavo H. B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2011
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3244463/
https://www.ncbi.nlm.nih.gov/pubmed/22216298
http://dx.doi.org/10.1371/journal.pone.0029504

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3244463/
https://www.ncbi.nlm.nih.gov/pubmed/22216298
http://dx.doi.org/10.1371/journal.pone.0029504

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