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Contact: John Ascenzi
ascenzi@email.chop.edu
267-426-6055
Children's Hospital of Philadelphia

Genome Editing Using Zinc Finger Nucleases (ZFNs)

Caption: From top, ZFNs on both sides of the DNA strand target a site upstream of the gene where most hemophilia-causing mutations reside. The ZFNs then cut both strands of the DNA double helix. As homologous recombination repairs the break in the strands, a normal copy of the clotting-factor gene (made from the donor template) is inserted into the DNA sequence, resulting in a corrected gene capable of expressing clotting factor. The mutated, nonfunctional gene remains in the DNA but is now harmless.

Credit: The Children's Hospital of Philadelphia

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Related news release: Genome editing, a next step in genetic therapy, corrects hemophilia in animals


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