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Now They See

Landmark Gene Therapy Restores Vision to Blind Children and Young Adults

Twenty-five children and young adults who had virtually no vision can now read several lines on an eye chart and see in a dimly lit setting thanks to an innovative gene therapy to cure a disease called Leber congenital amaurosis (LCA) that causes severe vision loss at birth. Considered a breakthrough in medical research, this gene therapy treatment has changed the lives of these young people and their families. One nine-year-old boy named Corey Haas has put away his white cane and is now playing little league baseball.

These individuals are participating in Phase I clinical trials of the treatment at The Children’s Hospital of Philadelphia and Moorfields Eye Hospital, as well as a Phase I clinical trial at the Universities of Pennsylvania and Florida. The Foundation Fighting Blindness, the leading private funder of retinal research in the world, has been a steadfast supporter of this research from the very beginning.



Historical Perspective
Historical Perspective

“I am overwhelmed with delight. We are providing vision to people who were virtually blind. This is the biggest advancement in the 38-year history of the Foundation Fighting Blindness,” says Gordon Gund, co-founder and chairman of the Foundation.

Gene Augmentation Therapy
Gene Augmentation Therapy

This particular form of LCA is caused by a defective gene called RPE65. The treatment involves delivery of a normal gene to the retina to augment function of the defective gene. The gene is delivered using a therapeutic virus known as an adeno-associated vector or AAV. Researchers believe the vision improvement from a single injection will last for many years.

Funding Future Treatments
Funding Future Treatments

The Foundation Fighting Blindness has been funding this research virtually every step of the way. Thanks to these recent advances in genetic research, clinical trials of gene therapy for other forms of retinal disease including retinoschisis, Stargardt disease, achromatopsia, and age-related macular degeneration are scheduled to begin shortly.


CHARITY PROFILE

Foundation Fighting Blindness

CFC NUMBER

11721

WHAT YOUR GIFT SUPPORTS

Your gift to the Foundation Fighting Blindness supports promising research into the development of treatments and cures for retinal degenerative diseases. Foundation-funded researchers are achieving remarkable success with a wide range of promising therapies including gene therapy, cell therapies, pharmaceutical and nutritional therapies and visual prosthetics. These and other sight-saving treatments are within our reach. With your support, a cure is in sight.