Story at a glance

  • Scientists used gene-editing to treat a patient who is blind due to a genetic disorder called Leber congenital amaurosis.
  • It could take up to a month to determine if the procedure was successful.
  • Researchers hope the procedure leads to promising treatment of other diseases.

Scientists for the first time have used a gene-editing technique called CRISPR inside someone’s body, in an effort to operate on DNA to treat a patient who is blind due to a rare genetic disorder. 

The companies that make the treatment, Editas Medicine and Allergan, announced Wednesday a patient recently had the procedure done at the Casey Eye Institute at Oregon Health & Science University in Portland. The procedure involves injecting the microscopic gene-editing tool into the eye of the patient, who is nearly blind from a condition called Leber congenital amaurosis.

Researchers say it could take up to a month to determine if and how well the procedure worked to restore the patient’s vision, but if all goes well, researchers plan to continue tests on 18 children and adults. 

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“We literally have the potential to take people who are essentially blind and make them see,” Charles Albright, chief scientific officer at Editas Medicine, told the Associated Press. “We think it could open up a whole new set of medicines to go in and change your DNA.” 

The condition is caused by a gene mutation that stops the body from making a protein necessary to convert light into signals to the brain, which allows for sight. 

The procedure’s aim is to edit or delete the mutation by making cuts on either side of it so the ends of DNA will reconnect and allow the gene to function properly. Researchers hope the procedure is an important step to bringing new treatments to patients with disease-causing gene mutations.

“This dosing is a truly historic event — for science, for medicine, and most importantly for people living with this eye disease,” Cynthia Collins, president and CEO of Editas Medicine, said in a statement. 

“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 us LCA10. We look forward to sharing future updates from this clinical trial and our ocular program.” 

The new study is the first to use CRISPR to edit a gene inside the body, but scientists have been using CRISPR to edit cells outside the body in efforts to treat cancer, sickle cell and other diseases. 

Published on Mar 04, 2020