RNA interference

doi:10.1136/bmj.328.7450.1245

From trainee to consultant, BMJ Group offers doctors around the world tailored information, special events, learning resources and recruitment services at every step along their career path.

... by doctors, for doctors, for patients
We are open for entries!

Online learning

BMJ Learning

High-quality CME / CPD for doctors and other healthcare professionals. BMJ Learning features hundreds of accredited, peer-reviewed learning modules in text, video, and audio formats. Find out more

Courses and Qualifications

BMJ Masterclasses

BMJ Masterclasses, led by experts, help clinicians to use the latest evidence and recent guidelines in practice and meet their CPD/CME requirements. Find out more

Exam Preparation

The leading provider of online exam preparation, helping over 167,000 healthcare professionals to pass their exams. Find out more

onExamination

Search all BMJ education articles:

From

Limit by

The BMJ iPad app brings you the best of print and online, including live links to the latest news, blogs, video, and podcasts. Get the BMJ iPad app.
You can use bmj.com to help you with your continuing medical education. Find out about CME/CPD credits for BMJ articles
Keep up to date with cardiology: Access the latest cardiovascular medicine resources from across BMJ Group.
Countdown to London 2012: BMJ Group's Olympics portal highlights latest Olympics and sports medicine-themed research, comment and learning

Access to the full text of this article requires a subscription or payment. Please log in or subscribe below.

Julian Downward, principal scientist (downward@cancer.org.uk)1

¹ Cancer Research UK London Research Institute, 44 Lincoln's Inn Fields, London WC2A 3PX

Introduction

Over the past decade “RNA interference” has emerged as a natural mechanism for silencing gene expression. This ancient cellular antiviral response can be harnessed to allow specific inhibition of the function of any chosen target genes, including those involved in causing diseases such as cancer, AIDS, and hepatitis. RNA interference is already proving to be an invaluable research tool, allowing much more rapid characterisation of the function of known genes. More importantly, the technology considerably bolsters functional genomics to aid in the identification of novel genes involved in disease processes. But can RNA interference be used as an effective therapeutic strategy? Many people in the biotechnology industry are betting that it can, but first considerable problems relating to delivery to target cells will have to be solved. This problem has proved the undoing of previous wonder technologies such as gene therapy and antisense. This review discusses the promises and pitfalls of RNA interference in research and treatment.

This article is based on a review of the literature on RNA interference and post-transcriptional gene silencing appearing in the PubMed database, along with personal experience of working in this field for the past four years. It also draws on consensus views expressed at several international conferences on RNA interference in 2003.

Current understanding of RNA interference

The first hints of the existence of the gene silencing mechanism that is now called RNA interference emerged from work on the genetic modification of plants in the late 1980s. Attempts to deepen the violet hue of petunias by expressing higher levels of an enzyme involved in the synthesis of the pigment unexpectedly resulted in the appearance of many white flowers. The introduction of extra copies of the gene had somehow caused a decrease in its expression rather than the anticipated increase.1 2

For some time this remained an unexplained …