Volume 87 Issue 39 | pp. 63-66
Issue Date: September 28, 2009

Targeting RNA

Unique challenges face developers of drugs that hit disease-related RNAs rather than disease-related proteins
Department: Science & Technology
News Channels: JACS In C&EN
Keywords: RNA, drug discovery, drug target

Developing drugs that target RNA continues to gain traction. Short RNA sequences that interfere with translation of messenger RNA are making their way through clinical trials. Now, drug discoverers are increasingly testing the idea that nonnucleotide small molecules that selectively target RNA sites might be therapeutically useful. The effort to discover such molecules with druglike properties is intensifying. But it isn’t easy.

“In medicinal chemistry and pharmaceuticals, the traditional targets are proteins, most commonly active . . .

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