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Eye Health Center

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Gene Therapy Helps People With Inherited Blindness See

Treating Two Eyes Safe and Effective, New Study Confirms

Half of Patients No Longer Legally Blind continued...

Morehouse says her vision became worse with each of her three pregnancies.

By the time she reached her early 40s, she saw little light or color, and most objects looked like “dark, hazy, blurry blobs,” she says.

The treatment involved injections of a genetically engineered virus that carried a normal version of the RPE65 gene.

After the first injections, the vision of six of the 12 study participants improved to the point that they were no longer classified as legally blind.

“One of the children who took part in the original study was riding a bicycle within a year,” says study co-author Manzar Ashtari, PhD, of the Children’s Hospital of Philadelphia. “This is a child who used a cane and held on to adults to guide him before having the treatment.”

Gene Therapy Might Prevent Blindness

The hope is that similar therapies targeting other mutations can be used to treat a large number of inherited diseases that cause blindness.

Katherine A. High, MD, of Children’s Hospital of Philadelphia, who also worked on the study, says there are now 200 known genetic mutations that cause vision loss.

Because LCA is a degenerative disease, there is also hope that the treatment may one day be used in very young children, or even babies, before vision loss has occurred.

“For many genetic diseases -- not just this one -- early intervention will hold the key to optimal outcomes,” High says.

Morehouse is most excited by this promise.

“If this treatment or treatments like this one can keep children from losing their vision in the first place and spare them the struggles that I had growing up, that is truly a miracle,” she says.

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