Design of a small molecule against an oncogenic noncoding RNA
Abstract
The goal of precision medicine is to identify selective drugs that modulate disease-causing biomolecules. This slow process often involves developing a high-throughput screen to test millions of potential drugs to find a few that affect the biomolecule. Here, we describe a facile approach using a disease-causing biomolecule's sequence to enable design of specific drugs, eliminating arduous and time-consuming screens. By using the sequence of a non-protein-coding, oncogenic RNA, we designed a drug specifically targeting the RNA's folded structure. In cells and animals, the drug inhibits its target, killing cancer cells while leaving healthy cells unaffected. Thus, a preclinical anticancer drug candidate can be quickly designed from sequence.
- Publication:
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Proceedings of the National Academy of Science
- Pub Date:
- May 2016
- DOI:
- 10.1073/pnas.1523975113
- Bibcode:
- 2016PNAS..113.5898V