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Gene therapy trial for cystic fibrosis

26 April 1999
By BioNews
Appeared in BioNews 5

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.

SOURCES & REFERENCES
US trial of gene therapy for cystic fibrosis
British Medical Journal |  23 April 1999
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