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Researchers have tested CRISPR gene editing inside a person’s body for the first time

Published on 05/03/20 at 11:11am
Photo by Caroline Davis

Allergan and Editas Medicine have treated their first patient in a clinical trial with CRISPR gene editing.

This therapy is being tested for treatment of Leber congenital amaurosis 10 (LCA10), an inherited type of blindness caused by mutations in the centrosomal protein 290 gene.

In the clinical trial, physicians at the Casey Eye Institute injected microscopic droplets that carried a hollowed-out virus engineered to deliver the genetic instructions to manufacture the CRISP gene-edits. This is a variation of gene therapy, where a preferable gene is delivered via a harmless virus.                                              

Cynthia Collins, President and Chief Executive Officer of Editas, said: “This dosing is a truly historic event—for science, for medicine, and most importantly for people living with this eye disease. The first patient dosed in the BRILLIANCE clinical trial marks a significant milestone toward delivering on the promise and potential of CRISPR medicines to durably treat devastating diseases such as LCA10. We look forward to sharing future updates form this clinical trial and our ocular program.”

CRISPR has been used before in a small number of patients with cancer, sickle cell anemia or beta-thalassemia. In those cases cells were removed the bodies of patients, edited using CRISP, then infused back into the patients. A controversial case saw Chinese researcher He Jiankui utilized CRISPR to alter the DNA of human embryos a pair of twins who were subsequently born, doing so outside the human body during in vitro fertilization.

Conor Kavanagh


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