Gene Therapy

CRISPR Gene Editing Performed for First Time in Patient’s body to Reduce Blindness

Posted on 9 March 2020

We literally have the potential to take people who are essentially blind and make them see

Charles Albright, the chief scientific officer at Editas Medicine

In an hour long procedure, undertaken at the Oregon Casey Eye Institute, three drops of fluid, containing genetically engineered viral vector, were injected into a patients eye, with the hope to restore the individuals sight.

Dr. Jason Comander, an inherited retinal disorder specialist at Massachusetts Eye and Ear in Boston, points to a model of an eye during an interview on Jan. 8.   (AP Photo/Rodrique Ngowi)

This gene editing procedure was aiming to knockout the CEP290 gene, thought to cause Leber congenital amaurosis. This is a rare, and previously incurable, genetic eye disorder causing blindness of the victim from birth.

One of the major benefits of this gene therapy is that if it is successful, it is permanent, the corrected cells will persist for life.

Although gene editing tools have been used in the body before, CRISPR is a much simpler tool for locating and cutting DNA at a particular site, with some scientists lauding this as “a new era in medicine”.

Andreas Lauer, M.D., right, performs the first-ever in vivo CRISPR gene edit procedure for the BRILLIANCE clinical trial. (OHSU/Kristyna Wentz-Graff)

There are currently 18 other patients lined up for a similar procedure if the first procedure is deemed a success after a waiting period.


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