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A sizable slice of the drug development pie is an exercise in targeting proteins. Find an active site or pocket on a problematic protein, stuff in a small molecule to interfere with that protein’s function, and, if all goes well, treat the disease caused by that malfunctioning macromolecule.
But only about 15 percent of proteins have such a convenient pocket or active site, leading the rest to be considered “undruggable.” Several researchers and drug developers are realizing that the solution might be to target disease-linked proteins at the RNA level instead (See “Scientists Take Aim at Disease-Causing RNAs Using Small-Molecule Drugs,” The Scientist, April 2019).
Messenger RNAs are one obvious target, because they encode proteins and even interfere with cellular processes directly in some diseases. But other RNAs are in some drug developers’ sights: noncoding RNAs, such as microRNAs that regulate gene expression, could be worth ...
