Doctors Edit Genes from Within the Human Body for the First Time, Treat Genetic Blindness

A gene-editing tool has for the first time been used to edit human genes inside the body – known as in vivo gene editing – to help repair the sight of people born with a blindness-causing gene mutation.

Clinicians from Oregon Health & Science University’s (OHSU) Casey Eye Institute in the U.S. used the CRISPR gene-editing tool for the BRILLIANCE clinical trial in an aim to repair a rare form of inherited blindness.

“Being able to edit genes inside the human body is incredibly profound,” says Dr. Mark Pennesi, who leads OHSU’s involvement in the trial. “Beyond potentially offering treatment for a previously untreatable form of blindness, in vivo gene editing could also enable treatments for a much wider range of diseases.”

Previous approaches have involved editing genetic material after it was removed from the body. But according to the announcement from trial sponsors Allergan plc and Editas Medicine, Inc., the first patient treated using in vivo  gene editing has shown promising results in lastingly curing such diseases.