The possibility of a genetic treatment for cystic fibrosis has moved a step closer with the start of a gene therapy trial at Stanford University Medical Center in California. The treatment involves the delivery, by aerosol, to the patient's lungs of a manufactured version of the cystic fibrosis transmembrane conductance regulator (CFTR) gene packed inside a virus shell. In cystic fibrosis, both copies of the CFTR gene are faulty. Cystic fibrosis is the most common life-threatening inherited disorder amongst the white populations of the UK and US, affecting around 7000 people in the UK and at least 60,000 people worldwide.
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Gene therapy and drug studies show promise for treatment of cystic fibrosis
Scientists have used two new techniques to fix defects in lung cells from people with cystic fibrosis, raising hope for new treatments for the disease in the future. The first study, published in the journal Plos Biology, used a gene therapy technique to treat the cells, whilst the second study, published in the American Journal of Respiratory Cell and Molecular Biology, used a drug called miglustat....
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