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FDA Approves Gene Therapy Treatment For Rare Form Of Blindness

FDA Approves Gene Therapy Treatment For Rare Form Of Blindness

Posted on: Tuesday, April 10, 2018
Author: Rebecca Posner, OD


As someone who continually works to assist patients with their vision here at Matossian Eye Associates, I am passionate about any medical breakthrough that could help individuals regain their sight. I can only begin to imagine how challenging it must be to go blind at any age, especially before the age of 18.

Philadelphia-based Spark Therapeutics has just launched a life-changing gene therapy treatment designed to combat a rare form of blindness. The mutation of a specific gene known as the RPE65 gene can cause visual disabilities, from birth, which can then lead to blindness by the age of 18. The retina (the portion of the visual system located at the back of the eye) eventually loses the ability to send light signals to the brain, and blindness occurs. This is known as retinal dystrophy.

Luxturna™ - The United States’ First FDA Approved Inherited Gene Therapy Treatment

In December of 2017, the FDA approved the nation's first inherited gene therapy treatment. Luxturna is an injectable treatment, although its application takes place as a surgical procedure. Luxturna contains a corrective form of the RPE65 gene, and it is applied directly to the retina cells, which allows them to resume production of a protein necessary for normal vision.

The good news is that clinical trials show significant and long-lasting improvement. In other words, the evidence seems to indicate that this is a long-term solution and offers hope for anyone suffering from this particular rare form of blindness. A total of 41 participants showed significant improvement navigating obstacles at low light levels compared to the control group who had not been given the treatment. Perhaps even more excitingly, some of the participants were able to see the moon, stars and even faces for the first time.

What Does This Mean For the Future of Inherited Genetic Therapy?

Another potentially exciting aspect to Luxturna is that there are about 220 different genes involved in the vision process. The hope is that this breakthrough will be the first of many in dealing with blindness and other vision problems.

The team at Matossian Eye Associates is dedicated to researching and delivering the best treatments for our patients. We keep up-to-date with all of the latest medical developments, in order to ensure that we can offer all of our patients the most advanced care and treatments possible.

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