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

Haifeng Geng; Grace Whiteley; Jameson Ribbens; Wei Zheng; Noel Southall; Xin Hu; Juan J. Marugan; Marc Ferrer; Gustavo H. B. Maegawa

doi:10.1371/journal.pone.0029504

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Peer-reviewed

Research Article

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

Haifeng Geng ,

Contributed equally to this work with: Haifeng Geng, Grace Whiteley

Affiliation McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, Maryland, United States of America
⨯
Grace Whiteley ,

Contributed equally to this work with: Haifeng Geng, Grace Whiteley

Affiliation National Institutes of Health, NIH Chemical Genomics Center, Rockville, Maryland, United States of America
⨯
Jameson Ribbens,

Affiliation McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, Maryland, United States of America
⨯
Wei Zheng,

Affiliation National Institutes of Health, NIH Chemical Genomics Center, Rockville, Maryland, United States of America
⨯
Noel Southall,

Affiliation National Institutes of Health, NIH Chemical Genomics Center, Rockville, Maryland, United States of America
⨯
Xin Hu,

Affiliation National Institutes of Health, NIH Chemical Genomics Center, Rockville, Maryland, United States of America
⨯
Juan J. Marugan,

Affiliation National Institutes of Health, NIH Chemical Genomics Center, Rockville, Maryland, United States of America
⨯
Marc Ferrer,

Affiliation National Institutes of Health, NIH Chemical Genomics Center, Rockville, Maryland, United States of America
⨯
Gustavo H. B. Maegawa

* E-mail: gmaegaw1@jhmmi.edu

Affiliations McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, Maryland, United States of America, Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, Maryland, United States of America
⨯

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

Haifeng Geng,
Grace Whiteley,
Jameson Ribbens,
Wei Zheng,
Noel Southall,
Xin Hu,
Juan J. Marugan,
Marc Ferrer,
Gustavo H. B. Maegawa

Published: December 21, 2011
https://doi.org/10.1371/journal.pone.0029504

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Colorimetric assays
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Fibroblasts
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Small molecules
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Fluorescence
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Cultured fibroblasts
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Lysosomes
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