Gene Therapy Offers Hope for Blindness

Better Blindness Treatments

About 3,000 Americans have Leber congenital amaurosis, and only about 10% of these people have the RPE65 gene mutation that would make them candidates for the genetic therapy being studied.

But similar treatments could be developed to treat other inherited retinal conditions, including some forms of macular degeneration, Goldberg notes.

"The potential is tremendous, he says."

The results could even been more dramatic if the treatment is deemed safe enough for use in children with inherited retinal disease, says Stephen Rose, PhD, who is chief research officer for the Foundation Fighting Blindness.

"These studies together show the power of this treatment," he tells WebMD. "We may be able to prevent blindness in children and restore functional vision in adults who have been blind for many years."