Overview

Over the past decade or so, researchers have begun to glimpse the rich framework of RNA-based regulatory mechanisms in cells. RNA interference and related mechanisms have already been extensively used as a tool to explore fundamental biological questions. Researchers—and pharmaceutical companies—have high hopes for their therapeutic use as well.

A June 10, 2008 meeting at the Academy addressed some aspects of these therapeutic opportunities. The symposium covered a wide range of topics that researchers should be aware of as they consider safe, practical, and effective delivery of oligonucleotides as drugs. Some talks focused tightly on the stability, targeting, and quantification of short interfering RNA (siRNA) delivery, while others addressed more generally the need to identify appropriate targets and avoid immune side effects. In many of these areas, the history of antisense technology, which largely failed to achieve comparably lofty goals, provides a cautionary tale, but also practical experience in delivering oligonucleotides.