Arkin et al. at the University of California, San Francisco provide a tremendously insightful look at the process of small-molecule screening as a way to discover new and innovative ways to treat existing genetic diseases. This review encompasses the entire process of small-molecule screening starting with conceptualization and experimental design to hit validation and follow-up. Arkin et al. highlight many potential uses for small-molecule screening and provide numerous examples where existing drugs were repurposed to treat current genetic diseases.
https://www.ncbi.nlm.nih.gov/pubmed/29799800
