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DNA scissors enhance gene therapy ...now available to all comers

jerry submitted, created time 5 months 1 week (sciencenow.sciencemag.org)

Imagine you're trying to find a cure for a disease, such as cystic fibrosis, which results from a single defective gene. Using traditional gene therapy, you'd infect the patient with a virus containing a good copy of the gene, hoping the DNA inserts into the person's genome in a spot that doesn't trigger cancer. "There's got to be a better way," you tell yourself--and there is. About a decade ago, researchers began developing a new strategy that relies on proteins called zinc finger nucleases that bind to a very specific place on a chromosome. The nucleases work like scissors, cutting the DNA at this precise location--say, the site of your defective gene--so that the gene can be repaired. The technique has worked in fruit fly, plant, and even human cells. But there's a hitch: The best zinc finger nucleases come from a company called Sangamo BioSciences, which until now has shared them only through chosen academic collaborations. We say "until now" because a separate consortium has found a way to produce high-quality zinc finger proteins, which will be available for about $200 per pool.

 

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