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Small-Molecule Screening for Genetic Diseases

25 May 2018

Abstract excerpt

The genetic determinants of many diseases, including monogenic diseases and cancers, have been identified; nevertheless, targeted therapy remains elusive for most. High-throughput screening (HTS) of small molecules, including high-content analysis (HCA), has been an important technology for the discovery of molecular tools and new therapeutics. HTS can be based on modulation of a known disease target (called...

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PMID: 29799800
DOI: 10.1146/annurev-genom-083117-021452

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