new gene therapy with a twist

[Guest guest]

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,â€

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.â€

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.

Becki

YOUR FAVORITE LilGooberGirl

YOUNGLUNG EMAIL SUPPORT LIST

www.topica.com/lists/younglung

Pediatric Interstitial Lung Disease Society

http://groups.yahoo.com/group/InterstitialLung_Kids/