First Successful Use of Genome Editing In Living Animals Cures Hemophilia In Mice

Monday, June 27, 2011 - 11:31 in Biology & Nature

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...

Read the whole article on PopSci

More from PopSci

Latest Science Newsletter

Get the latest and most popular science news articles of the week in your Inbox! It's free!

Check out our next project, Biology.Net