Gene Therapy Timeline

Produced by Nowgen, with support from the Wellcome Trust, this series of video clips describe some of the research milestones that have taken place in the development of gene therapy as a possible treatment for cystic fibrosis (CF).

The short video clips cover:

Introduction
Introduces Dr Kevin Southern, who is a consultant working with children who have cystic fibrosis

Discovering the CFTR gene
Dr Southern decribes the discovery of the faulty gene responsible for cystic fibrosis. This led to the idea of replacing this gene with a normal gene as a potential therapy.

Development of viral vectors to deliver genes
This clip looks at how, in the 1990's, viruses were investigated as a way of getting the normal cystic fibrosis gene into patients' airways.

The cystic fibrosis mouse
The cystic fibrosis mouse has been genetically modified to have cystic fibrosis. This animal model has been used to test possible gene therapies.

Testing new gene on adults
In 1997, studies began on looking at treating adults with cystic fibrosis by gene therapy.

Using liposomes to deliver genes
Liposomes are small lipid spheres that can be used to carry genes during gene therapy. These are being investigated as an alternative to using viruses to carry the genes.

The Gene Therapy Consortium
Dr Southern describes the collaboration between a number of institutions working on gene therapy for cystic fibrosis.

• URL: http://stem.org.uk/rx7v7