Nature opens new doors: RNA can be targeted by small molecule drugs!

Currently, nearly all available drugs target one of about 700 disease-related proteins out of the roughly 20,000 human proteins identified by the Human Genome Project

"These noncoding RNAs play a very important role in the genome, and we now know that mutations in this noncoding space can lead to disease," said Jeannie Lee, senior author of the Nature paper and MGH's Department of Molecular Biology.

However, the pharmaceutical industry has historically been hesitant to target RNA as a drug

Lee leads a molecular biology laboratory at MGH, where she and her team study RNA and its role in a biological process known as X chromosome inactivation (XCI)

Together with Merck scientists Kerrie Spencer and Elliott Nickbarg, the MGH team screened Xist from a library of 50,000 small-molecule compounds and found that several bound to the Repeat a (RepA) region on Xist

The method employed in this study can be used to identify other RNA-targeting drugs

Journal Reference :

1. Rodrigo Aguilar, Kerrie B.