Scientists have made the first attempt to edit a man’s genes inside his body, permanently changing his DNA to try to cure a metabolic disease called Hunter syndrome.
They hope the technique can one day be applied to other conditions including hemophilia and sickle cell disease, according to The Associated Press.
“We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending,” said Sandy Macrae, MB, ChB, PhD, president of Sangamo Therapeutics, the California company testing this for two metabolic diseases and hemophilia. “It becomes part of your DNA and is there for the rest of your life.”
As with any new therapy, there may be risks that haven’t been uncovered yet, but outside expert Eric Topol, MD, said studies should continue because they are targeting incurable diseases.
The AP highlights some of the ways in which this technique differs from other forms of gene therapy in the story below:
Daniel joined TriMed’s Chicago editorial team in 2017 as a Cardiovascular Business writer. He previously worked as a writer for daily newspapers in North Dakota and Indiana.