First Successful Use of Genome Editing In Living Animals Cures Hemophilia In Mice
Lab Mouse Wikimedia Commons A targeted snip through DNA's double helix can take out a mutated gene that causes hemophilia, curing mice of the disease, a new study found. It's the first study to use this form of genome editing in a living animal, and it could have implications for genetic treatment of other diseases, notably AIDS. Scientists say the research is a major step forward for gene therapy, which has long promised to cure disease by editing genetic sequences. The therapy is based on enzymes called zinc-finger nucleases, which serve as a sort of genetic scissors. The enzymes are engineered to match a specific gene location on a chromosome, where they snip through DNA's double helix. In this case, researchers led by Katherine A. High, a hematologist and gene therapy expert at The Children's Hospital of Philadelphia, used ZFN proteins that were engineered to snip through the location of a genetic...