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OHSU to provide new, FDA-approved gene therapy for a form of blindness

Oregon one of four places nationwide currently set to offer treatment for this kind of inherited retinal disease
Eye exam
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Oregon Health & Science University will be one of just four institutions nationwide to initially provide a new gene therapy treatment for a form of blindness that affects both children and adults.

The treatment - called Luxturna and developed by Spark Therapeutics of Philadelphia - was approved by the Food and Drug Administration on Dec. 19 for patients 12 months and older, making it the first approved gene therapy for an inherited disease in the United States. Luxturna has been shown to improve visual function in children and adults with inherited retinal disease caused by mutations in a gene called RPE65.

OHSU’s Casey Eye Institute plans to start providing the treatment sometime in 2018. Only three other institutions are currently slated to provide the treatment. More information about where treatments will be offered will be online at

“Treatments for childhood blindness have profound effects because these children have their whole lives ahead of them,” said David Wilson, M.D., director of Casey Eye Institute. “OHSU is proud to offer this treatment to help our patients keep their sight.”

The newly approved treatment involves injecting a modified virus into a patient’s eyes to correct a genetic mutation in the RPE65 gene. That mutation prevents prevents the production of a protein needed in the retina, a light-sensitive tissue in the back of the eye that enables people to convert light into signals that the brain interprets as images.

“This marks a turning point in treating rare inherited retinal diseases,” said Mark Pennesi, M.D., Ph.D., chief of the ophthalmic genetics division at Casey Eye Institute. “There are over 250 different genes that can cause these types of diseases and the fact the gene therapy can work for one, indicates that many more may be treatable by this approach.”

In the interest of ensuring the integrity of our clinical care practices and as part of our commitment to public transparency, OHSU actively regulates, tracks and manages relationships that our clinicians may hold with entities outside of OHSU. In regards to this treatment, Dr. Pennesi has received payments for consulting from Spark Therapeutics. Review details of OHSU's conflict of interest program to find out more about how we manage these business relationships.

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