RNA (ribonucleic acid) plays many roles in human health, and now a study in the journal Nature offers powerful evidence that RNA could also be a viable target for drug development. This work, led by researchers at Massachusetts General Hospital (MGH), suggests that a new class of biological factors numbering in the thousands can be targeted and thereby heralds a new era in drug development.
Nearly all drugs currently available target one of approximately 700 disease-related proteins among the roughly 20,000 human proteins identified by the Human Genome Project. However, in recent years there has been growing interest in expanding the list of “druggable” targets to include RNA. In cells, DNA (deoxyribonucleic acid) carries the genetic code for forming proteins. A segment of DNA is copied, or transcribed, into a “coding” RNA, which is in turn translated into protein. However, the vast majority of RNA in the human genome—98 percent—is “noncoding.”
