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.
Gene therapy trial for cystic fibrosis
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
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...
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