Just Gene Therapy is a not for profit organisation established to raise funds for gene therapy for cystic fibrosis.

Cystic fibrosis

Cystic fibrosis (or CF) is a life-threatening inherited condition, which affects the lungs, the digestive system and other organs, by interfering with the correct formulation of mucus. As a result, a build up of thick, sticky mucus prevents these organs from working properly. 90% of the deaths which are caused by cystic fibrosis are from lung failure.

Current treatment for cystic fibrosis is focused on control of the symptoms including regular and aggressive use of antibiotics, physiotherapy, digestive enzyme tablets and many other drugs. The everyday regime of a person with cystic fibrosis is dominated by the need for twice or thrice daily treatment, which may initially take an hour or two each day and which increases to four, six or more as the condition worsens. Regular hospital appointments are the norm and for those with particular infections and for those who are sicker, long frequent stays in hospital are often necessary.

The burden of having cystic fibrosis and the necessary treatment to stay alive cannot be overstated. Not all families are able to cope and teenagers in particular often rebel and refuse to comply, to the anguish of their families, the despair of their medical team and at detriment to their own health.

Gene therapy offers a realistic prospect of breaking into this cycle of gradual decline and ever-increasing burden of treatment by tackling the disease at its root – the genetic cause of the condition.

CF continues to claim two to three young lives each week in the UK and many more throughout the world, but a real breakthrough is possible and indeed likely within the foreseeable future.

 

 

Mucus on the cilia in the lungs of someone with CF.

Mucus on the cilia in CF lungs.


A person with CF measuring her lung function

A person with CF measuring her lung function.

© Just Gene Therapy 2012-14 | Just Gene Therapy is raising money on behalf of Imperial College, an exempt charity*.