PerspectiveMolecular Biology

A splicing magic bullet

Science  08 Aug 2014:
Vol. 345, Issue 6197, pp. 624-625
DOI: 10.1126/science.1258444

You are currently viewing the summary.

View Full Text

Via your Institution

Log in through your institution

Log in through your institution


Summary

At the turn of the 20th century, the German physician Paul Ehrlich championed the concept that chemicals with special affinities for particular pathogens could serve as “magic bullets” to combat infections with no side effects on the host. In this vein, he went on to discover arsphenamine, an effective antisyphilis drug (1). On page 688 of this issue, Naryshkin et al. (2) identify compounds that selectively modulate the splicing of a particular mRNA precursor, offering potential new treatments for spinal muscular atrophy (SMA), the most common genetic cause of infant death (3). Gene-specific RNA-processing drugs could provide unique tools for studying gene function and present a new therapeutic approach for other diseases as well.