New breakthrough could help design drugs from genome sequence

Researchers including an Indian-origin have developed a potentially general approach to design drugs from genome sequence.

Sai Pradeep Velagapudi, the first author of the study and a graduate student working in the Disney lab, said that with their program, they can identify compounds with high specificity, asserting that in the future, they hope they can design drug candidates for other cancers or for any pathological RNA.

Discovered only in the 1990s, microRNAs are short molecules that work within virtually all animal and plant cells.

In the new study, Matthew Disney, PhD, an associate professor at The Scripps Research Institute (TSRI) ,who led the study and team, describe their computational technique, which identifies optimal drug targets by mining a database of drug-RNA sequence ("motif") interactions against thousands of cellular RNA sequences.

Using Inforna, the team identified compounds that can target microRNA-96, as well as additional compounds that target nearly two dozen other disease-associated microRNAs.

The researchers showed that the drug candidate that inhibited microRNA-96 inhibited cancer cell growth. Importantly, they also showed that cells without functioning microRNA-96 were unaffected by the drug.

Disney added that the new drug candidate, which is easy to produce and cell permeable, targets microRNA-96 far more specifically than the state-of-the-art method to target RNA (using oligonucleotides) currently in use.

The technique has been published in the journal Nature Chemical Biology online.