Treatment for Inherited Vision Loss

Gene therapy is a technique that is used to treat and prevent genetic diseases. This method introduces new genes into cells to take the place of missing or defective genes. There were many notable developments in the area of gene therapy this year. Specifically, the first in vivo gene therapy, Spark Therapeutics’ Luxturna, was recently approved in the U.S.

What is In Vivo Gene Therapy?

In vivo therapy is when genes are inserted into cells directly in a patient’s body, rather than in the lab.

Treatment for Patients with Inherited Vision Loss

Luxturna is intended to treat patients with Leber congenital amaurosis (LCA), a rare vision disease caused by a gene defect.

This treatment has the potential to significantly improve vision for LCA patients. According to Rob Stein, Correspondent and Senior Editor of NPR's Science Desk:

"In tests on patients, the treatment often produced dramatic results, restoring the ability of patients to see things they could never see before, such as the stars, the moon, fireworks and their parents' faces. The treatment also enabled patients to do many things that had been impossible, such as read, play sports, ride bicycles and go outside at night by themselves."

To learn more about this breakthrough, view the FDA News Release.

This post originally appeared on the Astarte Biologics blog and has been modified for Patch.