A new “find-and-replace” genome editing method enables scientists to make large-scale changes to the genetic code of a living cell, faster than previous editing technology by a factor of two. The new method could be used to engineer cells that produce new proteins, or to design genetic “firewalls” that would prevent engineered cells from spreading their DNA.
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.