Validating therapeutic targets through human genetics, Robert M. Plenge, Edward M. Scolnick and David Altshuler. Nature Reviews Drug Discovery. 2013 Aug;12(8):581-94. doi: 10.1038/nrd4051. Epub 2013 Jul 19. PMID: 23868113
We describe a strategy to use human genetics as a powerful tool to validate therapeutic targets for drug discovery. We frame the concept of “genotype-phenotype dose-response curves”.