Gene Therapy Helps People With Inherited Blindness See
Treating Two Eyes Safe and Effective, New Study Confirms
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.