Phenotypic screens for compounds that target the cellular pathologies underlying Parkinson's disease
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 embr...
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| Format: | Article |
| Language: | en_US |
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Elsevier
2016
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| Online Access: | http://hdl.handle.net/1721.1/101272 https://orcid.org/0000-0003-1307-882X |
| _version_ | 1811000639807815680 |
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| author | Tardiff, Daniel F. Lindquist, Susan |
| author2 | Massachusetts Institute of Technology. Department of Biology |
| author_facet | Massachusetts Institute of Technology. Department of Biology Tardiff, Daniel F. Lindquist, Susan |
| author_sort | Tardiff, Daniel F. |
| collection | MIT |
| description | 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. |
| first_indexed | 2024-09-23T15:19:57Z |
| format | Article |
| id | mit-1721.1/101272 |
| institution | Massachusetts Institute of Technology |
| language | en_US |
| last_indexed | 2024-09-23T15:19:57Z |
| publishDate | 2016 |
| publisher | Elsevier |
| record_format | dspace |
| spelling | mit-1721.1/1012722022-10-02T02:15:46Z Phenotypic screens for compounds that target the cellular pathologies underlying Parkinson's disease Tardiff, Daniel F. Lindquist, Susan Massachusetts Institute of Technology. Department of Biology Whitehead Institute for Biomedical Research Lindquist, Susan 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. 2016-02-25T14:18:16Z 2016-02-25T14:18:16Z 2012-03 Article http://purl.org/eprint/type/JournalArticle 17406749 http://hdl.handle.net/1721.1/101272 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. https://orcid.org/0000-0003-1307-882X en_US http://dx.doi.org/10.1016/j.ddtec.2012.02.003 Drug Discovery Today: Technologies Creative Commons Attribution-Noncommercial-NoDerivatives http://creativecommons.org/licenses/by-nc-nd/4.0/ application/pdf Elsevier PMC |
| spellingShingle | Tardiff, Daniel F. Lindquist, Susan Phenotypic screens for compounds that target the cellular pathologies underlying Parkinson's disease |
| title | Phenotypic screens for compounds that target the cellular pathologies underlying Parkinson's disease |
| title_full | Phenotypic screens for compounds that target the cellular pathologies underlying Parkinson's disease |
| title_fullStr | Phenotypic screens for compounds that target the cellular pathologies underlying Parkinson's disease |
| title_full_unstemmed | Phenotypic screens for compounds that target the cellular pathologies underlying Parkinson's disease |
| title_short | Phenotypic screens for compounds that target the cellular pathologies underlying Parkinson's disease |
| title_sort | phenotypic screens for compounds that target the cellular pathologies underlying parkinson s disease |
| url | http://hdl.handle.net/1721.1/101272 https://orcid.org/0000-0003-1307-882X |
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