The aliens are coming -- and they be us!

[Guest guest]

So, how long before humans are diverse enough to look like the the

aliens in Star Wars?

 

 

-

" The Havens " <thehavens

<Recipient list suppressed>

Friday, January 04, 2002 8:53 AM

Gene therapy hope for cystic fibrosis

 

 

> SMaRT treatment for fatal disease shows promise.

> 4 January 2002

> JEREMY THOMSON

>

> A new form of gene therapy that fools the body into repairing the faulty

> gene in cystic fibrosis sufferers is looking promising in the lab.

>

> Xiaoming Liu and colleagues at the University of Iowa, Iowa City, used a

> technique dubbed 'SMaRT' to sneak a decoy genetic sequence into diseased

> human lung cells grafted into a living mouse. The cells' function was

> boosted by 10 per cent - enough to keep the disease under control1.

>

> Cystic fibrosis, the most common life-threatening genetic illness in

Europe

> and the United States, prevents salts from moving through body tissues

> properly, disrupting digestion and more importantly leading to chronic

lung

> infection. Despite great advances in treatments, many patients do not

reach

> adulthood and no cure is available.

>

> For the past decade researchers have had little success treating cystic

> fibrosis with conventional gene therapy, in which a complete corrected

gene

> is simply added to the patient's DNA. SMaRT works differently. It

> interferes with the processing of RNA - the intermediate stage between DNA

> and the proteins that make up body tissue.

>

> When the body constructs a new protein, it first copies the relevant DNA

> sequence into a blueprint RNA strand. Mammals' DNA contains long stretches

> of 'junk', copies of which must be removed from RNA before the final

> protein can be built. So a cellular machine called the ribosome snips the

> RNA strand into pieces, discards the junk and stitches it together again.

>

> Liu's team take advantage of the ribosome's tidying to sneak in a new RNA

> fragment. As the ribosome pastes the snipped-up RNA chunks back together,

> it incorporates the new sequence, which corrects the error that causes

> cystic fibrosis in the cell.

>

> The researchers engineered a virus to infect lung cells and implant the

> fragment - called a 'minigene' - into their DNA. As it is read at the same

> time as the faulty cystic fibrosis gene, the minigene is always present in

> the cell when the ribosome is doing its cutting and pasting.

>

> Because the technique acts at the intermediate RNA stage, the cell still

> regulates the expression of the gene. This ensures that the right amount

of

> protein is produced in the right places - a big advantage over

conventional

> gene therapy.

>

> Despite its promise, the technology has a long way to go before it reaches

> the clinic. The viruses that deliver the minigene are not very efficient,

> and the body's immune system soon destroys them.

>

> Another problem is persuading the cell to keep using the new minigene.

>

> " Fooling Mother Nature is not easy, " says Ronald Crystal, a gene-therapy

> expert at Cornell University in Ithaca, New York. " The central dogma of a

> gene begetting the protein it encodes is something that the cell defends

> with a vengeance. "

>

> References 1. Liu, X. et al. Partial correction of endogenous F508 CFTR in

> human cystic fibrosis airway epithelia by spliceosome-mediated RNA

> trans-splicing. Nature Biotechnology, 20, 47 - 52, (2002).

> © Nature News Service / Macmillan Magazines Ltd 2002

>

>

>

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