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Human Genes Corrected

  April, 5 2005 10:02
your information resource in human molecular genetics
With a discovery published online in Nature that could herald an alternative to gene therapy, Michael Holmes and his colleagues demonstrate a new way to correct disease-causing mutations in human DNA.

Researchers have struggled to perfect gene therapy, in which they try to compensate for defective genes by inserting a new working copy into cells. Gene correction takes a different approach: repairing the mistakes in DNA that cripple the gene.

Holmes' team fused a zinc-finger protein, which recognizes three to four base pairs in DNA, to a nuclease, which cuts DNA at that sequence. These 'zinc-finger nucleases' are known to trigger homologous recombination, a process by which the surrounding segment of DNA is replaced by a fresh copy.

The team designed a zinc-finger nuclease that recognizes the mutation underlying X-linked severe combined immune deficiency (SCID) - children with this condition can suffer life-threatening infections soon after birth. Holmes and co-workers showed that the nuclease prompted correction of the defect in a high frequency of T cells - the cells affected by the disorder. The study offers the prospect that zinc-finger nucleases could be engineered against any disease-causing mutation in the human genome, and used to treat the disease.

Author contact:

Michael Holmes (Sangamo, BioSciences, Richmond, CA, USA)
E-mail: mholmes@sangamo.com

(C) Nature press release.

Message posted by: Trevor M. D'Souza

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