AAV cystic fibrosis gene therapy
on 16/02/09
 BBC News |
Cystic fibrosis gene cure closer
BBC News, UK - 17 hours ago Around 8,000 people in the UK have cystic fibrosis and one in 25 are carriers for the condition. CF produces thick, sticky mucus that clogs the lungs and the pancreas, leading to life-threatening chest infections and problems with digestion. Existing treatments only ease symptoms and life expectancy is low - most with CF die before they reach 40. In the future it is hoped that gene therapy will provide a cure by replacing the defective CF gene which disables or destroys a protein known as CFTR. A University of California team, working with experts from the University of Iowa, mutated the adeno-associated virus to develop two major features - the ability to bind to different receptors or bind to a more plentiful receptor on the surface of lung cells and to make it past the cell surface membrane and into the cell. In human lung tissue, it completely rescued the chloride ion transport properties of the cells after delivering the correct copy of the CFTR gene to replace the mutated copy of the gene that is present in CF patients. They now plan to test the treatment further and to adapt the virus to gene therapy for other diseases, including Alzheimer's. Cystic fibrosis sufferers offered hope by gene therapy treatment Telegraph.co.uk Zombie Watch: Curing Cystic Fibrosis With Modified Viruses Daily Californian Cure for cystic fibrosis 'a step closer' Marie Claire.co.uk Local Edition all 13 news articles |
