iPlayer Radio What's New?
Image for 20/06/2012

Listen now 28 mins

Listen in pop-out player


28 minutes
First broadcast:
Wednesday 20 June 2012

Gene therapy - repairing malfunctioning cells by mending their DNA - offers an elegant solution to diseases, such as cystic fibrosis, caused by a single flawed gene. It's a very simple concept to describe - simply insert a 'normal' gene to do the job - but it's this process, the delivery of the gene, that's proving to be so difficult and time consuming. Since the first human study began in 1990 the field has struggled with various technical challenges and set-backs. But over a decade on, researchers are beginning to report successes in treating several devastating diseases. Geoff Watts finds out about some of the new techniques for gene therapy, and discovers how these are now being used in a trial of a new method of gene therapy for cystic fibrosis.

12 years ago, a group of scientists from Imperial College in London, Oxford and Edinburgh formed the Cystic Fibrosis Gene Therapy Consortium. This year they started the world's biggest trial of gene therapy for cystic fibrosis. Funded by the Cystic Fibrosis Trust, the Medical Research Council and The National Institute for Health Research , the trial will treat 120 CF patients with either a placebo or a healthy copy of the gene that causes CF. The gene is wrapped up in a fat globule, or liposome and delivered in aerosol form directly to the lungs.

Producer: Fiona Roberts.


Plants: From Roots to Riches

Plants: From Roots to Riches

Our relationship with plants: a major new history by Kew's science director Kathy Willis

Inside Science

Image for BBC Inside Science

Adam Rutherford explores the research that is transforming our world.

Free download

  1. Image for Frontiers


    Frontiers explores new ideas in science and meets the people behind them. There are two six-part…

BBC © 2014 The BBC is not responsible for the content of external sites. Read more.

This page is best viewed in an up-to-date web browser with style sheets (CSS) enabled. While you will be able to view the content of this page in your current browser, you will not be able to get the full visual experience. Please consider upgrading your browser software or enabling style sheets (CSS) if you are able to do so.

Added. Check out your playlist Dismiss