First test of in-body gene editing shows promise

Monday, September 10, 2018

Nature, by Heidi Ledford
Preliminary results suggest that treatment for rare disease is safe, but its effectiveness is unclear. 

A therapy that edits genes directly in the human body might be safe, suggest early findings from the first trial to test the approach.

Researchers from Sangamo Therapeutics in Richmond, California, designed enzymes to correct an error in the genome of people with a rare genetic disease called Hunter syndrome, who cannot break down some complex sugars. The scientists used a virus to deliver these gene-editing enzymes into the cells of patients. Read more.