RE: [cfparentsBECKI

[Guest guest]

Becki,

I heard Dr Parson's on the radio today. He mentioned that it was a PHd

student who discovered this and she was in the US at the moment as she

has been offered a position to further this study and lots of genetic

institutes. He said it would take 5-10years before patient trials. He

also said they need funding.

(Australia)

Gene Therapy Article

Sunday, 20 October, 2002, 23:04 GMT 00:04 UK

Gene therapy targets cystic fibrosis

Cystic Fibrosis is caused by a single defective gene

Scientists believe they may be able to overcome a natural body defence

which

is

hindering gene therapy for cystic fibrosis.

Thousands in the UK are battling the lung disease, which,

without transplantation, can prove fatal before the age of 30.

Gene therapy is one of the big hopes for future

treatments, as

a mutation in just one gene causes a malfunction within cells on the

lung

surface.

The malfunction leads to overproduction of mucus, which

clogs

the lung and makes sufferers vulnerable to chronic infections.

Because the surface of the lung is relatively accessible

compared with other internal organs, it is hoped that a gene therapy

could

be

inserted into an inhaler to replace the mutated gene.

However, trials of gene therapy have only had limited

success so far, with the effects often proving short-lived.

This is partly due to the natural defences of the lungs,

which

are primed to defend their surface against all sorts of allergens, dust

particles, viruses and other foreign particles.

This defence system proves pretty effective against the

viruses used to deliver the new gene into cells.

Keeping therapy working

Now a team of researchers from the Women's and Children's

Hospital in Adelaide, Australia, claim they have found a way of

prolonging

the

effect of the treatment.

As well as the therapy, they gave a dose of a

detergent-like

substance found naturally in the

healthy lung.

They believe this helps " condition " the surface of the

lung

and hold off the defence mechanism.

So far, they have only tested their theory in the nasal

passages of mice - which react in a similar way to the surfaces of the

lungs.

In their experiments, cells lining these airways not

only

responded to the gene therapy, but maintained that response - so far for

110

days.

As nasal cells are generally replaced once every three

months, this means it is possible that the change is appearing in

newly-created cells as well as existing ones.

Helpful HIV

The team used a modified form of HIV to carry the

therapy -

it has been rendered incapable of causing an HIV infection, but can

still

enter cells and replace the key gene with a new, healthy sequence.

Dr Parsons, who led the research, said: " Airway

cells

are replaced every three months so our findings are particularly

exciting

because they imply we are in fact targeting airway stem

cells

through this approach.

" Some of the therapeutic gene must have been passed on

from

these parent stem cells to their daughter cells for the effect to

persist

beyond three months. "

The next challenge is to take these successes and

reproduce

them in the lungs of real cystic fibrosis patients, and the Australian

team is fine-tuning the technique so that human clinical trials can be

started.

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/