First In Vivo Human Genome Editing to Be Tested in New Clinical Trial
By Abby Olena,
The Scientist
| 05. 18. 2017
Sangamo Therapeutics will use zinc finger nucleases to introduce the gene for a missing clotting factor into the livers of men with hemophilia B.
Researchers have edited the human genome before, but always in cells outside the body. Now, biotech company Sangamo Therapeutics is recruiting participants for clinical trials in which patients with hemophilia B, Hurler syndrome, or Hunter syndrome will have the gene coding for one of the enzymes that is non-functional in them stitched into their genomes at double-stranded DNA breaks caused by zinc finger nucleases.
“This is the first time someone could have a new gene put into their liver,” Sangamo President and CEO Sandy Macrae told The Scientist. “It’s a privilege and a responsibility to do” these trials.
One of the diseases Sangamo will focus on is hemophilia B, which is a severe bleeding disorder caused by a defective or absent gene called F9. The gene’s protein product, coagulation factor IX, is an enzyme essential for blood clotting.
Two other clinical trials will focus on Hurler and Hunter syndromes. People with these...
Related Articles
By Mike McIntire, The New York Times | 01.24.2026
Genetic researchers were seeking children for an ambitious, federally funded project to track brain development — a study that they told families could yield invaluable discoveries about DNA’s impact on behavior and disease.
They also promised that the children’s sensitive...
By Arthur Lazarus, MedPage Today | 01.23.2026
A growing body of contemporary research and reporting exposes how old ideas can find new life when repurposed within modern systems of medicine, technology, and public policy. Over the last decade, several trends have converged:
- The rise of polygenic scoring...
By Danny Finley, Bill of Health | 01.08.2026
The United States Food and Drug Administration (FDA) has a unique funding structure among federal scientific and health agencies. The industries it regulates fund nearly half of its budget. The agency charges companies a user fee for each application
...
By George Janes, BioNews | 01.12.2026
A heart attack patient has become the first person to be treated in a clinical trial of an experimental gene therapy, which aims to strengthen blood vessels after coronary bypass surgery.
Coronary artery bypass surgery is performed to treat...
