You are here:  Home > Human Health > Genes & health > Gene therapy
University of Bristol
Wellcome Trust
Recommended by:
Society of Biology
PEEP for Physics & Ethics at GCSE

Genes and health

Gene therapy

Gene therapy research aims to provide a replacement gene or gene sequence to do the job that the faulty gene is not doing. For example in Cystic Fibrosis a pair of faulty genes, one on each copy of chromosome 7, means that the body cannot make fully functioning CFTR (Cystic Fibrosis Transmembrane Conductance Regulator), which transports salt and water in and out of the cells that line the lungs and digestive system. The result is that the mucus lining the lungs and digestive system is very thick and sticky, and builds up causing respiratory and digestive problems. Gene therapy research is focussing on providing working copies of the CF gene to the lungs, as respiratory failure is the greatest threat to CF patients. However, gene therapy is proving much harder to implement in practice than it may sound in theory. The research is also expensive.

However, some faulty genes are not inactive, they actually produce a problem. For example in Huntington's Disease, a fault on chromosome 4 causes a variant copy of the protein, Huntingtin, to be produced. This builds up in the body and is toxic. So research into possible treatments for this and similar conditions has focused on ways of ‘switching off’ the faulty gene. In January 2005 in Nature Medicine a report from Iowa University claimed to have successfully switched off the gene that causes a similar brain disease in mice. The researchers hope to go on to trial a similar technique in humans, but estimate they are at least 5 years away from a potential treatment for humans that could be offered therapeutically. This may well be an optimistic estimate.

Next Ethical issues


What's your opinion?

Average rating

Not yet rated

Read comments

speech bubble  No comments yet. Why not be the first person to add one?

Bookmark this pagefacebook myspace bebo delicious diigo stumbleupon twitter reddit yahoo google


The research into switching off harmful genes in humans relies on research into similar conditions in other animals.

What are the ethical issues arising in the use of animals in such research?

Do you have a view on the use of animals in this way? Are you more influenced by your heart or your head?

Is the view of someone directly affected by the disease more or less valid than anyone else’s?