Phenotypic screens for compounds that target the cellular pathologies underlying Parkinson's disease
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Lindquist_Phenotypic screens.pdf
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703.51 KB
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Author(s) •
Tardiff, Daniel F.
Lindquist, Susan
Date Issued
March 2012
Journal
Drug Discovery Today: Technologies
Publisher
Elsevier
Citation
Tardiff, Daniel F., and Susan Lindquist. “Phenotypic Screens for Compounds That Target the Cellular Pathologies Underlying Parkinson’s Disease.” Drug Discovery Today: Technologies 10, no. 1 (March 2013): e121–e128.
Version
Final published version
Abstract
Parkinson's disease (PD) is a devastating neurodegenerative disease that affects over one million patients in the US. Yet, no disease modifying drugs exist, only those that temporarily alleviate symptoms. Because of its poorly defined and highly complex disease etiology, it is essential to embrace unbiased and innovative approaches for identifying new chemical entities that target the underlying toxicities associated with PD. Traditional target-based drug discovery paradigm can suffer from a bias toward a small number of potential targets. Phenotypic screening of both genetic and pharmacological PD models offers an alternative approach to discover compounds that target the initiating causes and effectors of cellular toxicity. The relative paucity of reported phenotypic screens illustrates the intrinsic difficulty in establishing model systems that are both biologically meaningful and adaptable to high-throughput screening. Parallel advances in PD models and in vivo screening technologies will help create opportunities for identifying new therapeutic leads with unanticipated, breakthrough mechanisms of action.
MIT Department
Massachusetts Institute of Technology. Department of Biology
Whitehead Institute for Biomedical Research
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Creative Commons Attribution-Noncommercial-NoDerivatives
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DOI of Published Version
https://doi.org/10.1016/j.ddtec.2012.02.003