Genetics and Health

DNA Spray for Cystic Fibrosis

by Hsien Hsien Lei, PhD on December 4th, 2006

A deletion of three base pairs in the gene for cystic fibrosis (CF), CFTR, is responsible for 70% of all CF cases. When the genetic mutation was first discovered, there was great hope that gene therapy could be developed that would “cure” cystic fibrosis. Unfortunately, that hasn’t happened yet.

Researchers at the Brompton in London and at the Western general hospital in Edinburgh are set to test a DNA spray for cystic fibrosis on 200 patients.

The technique being tested involves the use of a nebuliser that injects a fine spray into the airways. Within the spray are tiny soap-like bubbles called liposomes containing DNA without the cystic fibrosis defect.

The liposomes fuse with the outer surfaces of cells lining the lungs and the DNA passes by natural processes into their nuclei.

Once installed, it is hoped the DNA will prompt the cells to make the essential protein lacking in cystic fibrosis sufferers.

Because of the constant replacement of cells in the body, the treatment would have to be taken repeatedly to sustain its effect.

Read more about my college classmates, Isabel and Anabel Stenzel, who both had cystic fibrosis and received lung transplants a few years ago. An October podcast with Isabel Stenzel-Byrnes is available at Cystic Fibrosis Podcast. So glad to hear they’re doing well!

The Sunday Times, December 3, 2006

Technorati Tags: genetics, genes, dna, cystic fibrosis, cf, isabel stenzel byrnes, anabel stenzel, dna spray, gene therapy, diseases, illness, health

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