Gene therapy for cystic fibrosis improves lung function

A research consortium involving investigators at the University of Oxford has developed a gene therapy for cystic fibrosis that has demonstrated some proof-of-concept results that are encouraging new work on a hopefully more effective approach. Working with liposomes, the group says they were able to get a small but significant improvement in lung function for patients by delivering a copy of the CFTR gene. Mutations in the gene cause the disease and now the scientists say that better dosing and delivery with a viral vector may prove much more effective, according to a report in New Scientist. "We only delivered about a teaspoon of the treatment, and the possibility of being able to increase the dose to see a greater effect in more patients is very exciting," says Oxford's Deborah Gill. Report | Article

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