Gene Editing Treats Blood Disease

Revising a dysfunctional gene in vivo for the first time, researchers successfully restore blood clotting in hemophiliac mice.

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WIKIMEDIA COMMONS, AARON LOGAN

Using precision DNA-cleaving enzymes called zinc finger nucleases (ZFNs) to replace a dysfunctional gene in vivo, researchers successfully restored nearly normal blood clotting in mice with the human blood disease hemophilia B.

The feat, published online yesterday (June 26) in Nature, represents the first time scientists have been able to use ZFN-enabled "genome editing" to permanently correct the DNA of cells within a living animal, and provides hope that the same technique may one day treat a wide range of human diseases.

"This is a significant extension of the zinc finger nuclease technology that could help human medicine," said Mario Capecchi, a professor of human genetics at the University of Utah and a 2007 Nobel Prize winner for gene-targeting discoveries that made possible today's profusion ...

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