Children’s Hospital discover a new approach to correct genetic defects

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Researchers at Children's Hospital discover a new approach to

correct genetic defects created: July 07, 2003 - 9:07 PM

updated: July 08, 2003 - 7:40 AM

Written by: Dr. Clements, posted by Web Producer Jeannie

Piper

DENVER - She's only 26 years old, but Aspen Arbuthnot realizes she

may already have lived most of her life.

Dr. Clements reports on a new gene therapy with a twist.

July 7, 2003.

" Every time my birthday comes around I'm glad to have lived another

year, but yet I know that it's one step closer to not being here any

more, " Arbuthnot said.

Life expectancy with cystic fibrosis is short.

" I believe it's 32 years old right now, so I have six years left, "

she said.

Cystic fibrosis causes leaky lungs, secretions too thick to cough

out, setting up serious and even fatal lung infections. It happens

in nose tissues too, an easy place to do a clever experiment.

Researchers at Children's Hospital in Denver have found a way to

make microscopic bee-bee-like DNA strands and launch them into cells

to correct genetic defects. The DNA is put into a nose spray, acting

as the medication, which is then absorbed into the body.

" Low and behold what we discovered was once we gave a large enough

dose of the new gene, the cells almost always would start to

function normally, " said Dr. Jeff Wagener of Children's Hospital.

The DNA corrected the genetic defect, temporarily though, for up to

21 days.

" I'm very, very excited about it, " said Arbuthnot. " I hope that

everything works out OK with it. "

The next step is clinical trials for the lungs.

Before the study, doctors tried using viruses to sneak DNA

corrections into lung cells. It worked, but the viruses made

patients sick. This new method is a much cleaner approach,

researchers say.