New Technique Cures Hemophilia in Mice

Using an innovative gene therapy technique called genome editing that hones in on the precise location of mutated DNA, scientists have treated the blood clotting disorder hemophilia in mice. This is the first time that genome editing has been done in a living animal and achieved clinically meaningful results. As such, it represents an important step forward in the decades-long scientific progression of gene therapy  whereby a genetic condition is treated by correcting a problematic DNA sequence.