Translating Human Genetics into Novel Drug Targets.

The most important promise of the human genome sequencing project is the identification of the genetic cause of devastating human diseases and the subsequent deliver of novel drug therapies to treat these diseases with high unmet medical need. In the last 10 years we have successfully identified hundreds of genetic loci associated with many traits and diseases. The translation of these findings into novel therapies is not straightforward and poses challenges that are usually overlooked in traditional gene mapping. This chapter describes some of the most common challenges and opportunities to use human genetics to identify and validate novel drug targets.