Coordinated by the UK
Cystic Fibrosis Gene Therapy Consortium, the two - year study involved 136 CF patients aged 12 years or older from across the UK.
The current U.K. trial is again using fats, or lipids, but the DNA includes a segment called a promoter that should make gene expression last longer, says Eric Alton of Imperial College London, who heads the UK Cystic
Fibrosis Gene Therapy Consortium.
Mice with the cystic
fibrosis gene don't seem to get serious lung diseases, as humans do.
Despite expectations of a rapid breakthrough, no cystic
fibrosis gene therapy trial so far has been able to show long - term clinical improvement.
It is a success that is built on 25 years... Continue reading Clinical trial success for
Cystic Fibrosis gene therapy: built on animal research
To screen for such drugs, scientists attach firefly luciferase DNA to the end of the cystic
fibrosis gene.
It is a landmark moment: 22 years after the cystic
fibrosis gene was discovered, some patients are about to receive a drug called Kalydeco to treat the defect that causes their lungs to clog up with sticky mucus.
Francis Collins of the University of Michigan, who was a co-discoverer of the cystic
fibrosis gene, told a working group on genetics that if it was not «very forward looking», society could be caught off guard by the rapid pace of progress.
Collins wants to avoid repetition of the confusion that followed the discovery in 1989 of the cystic
fibrosis gene.
This morning the Cystic
Fibrosis Gene Therapy Consortium (GTC) announced the results of clinical trial in 140 patients with cystic fibrosis, which demonstrate the potential for gene therapy to slow — and potentially halt - the decline of lung function in people with the disorder.
«Now we are closer to understanding why patients with the exact same genetic mutation in the cystic
fibrosis gene have such widely varying manifestations of lung disease, and closer to finding new therapies.»
Phrases with «fibrosis gene»