Gene therapy successful and safe in restoring vision

Researchers at the University of Pennsylvania have used gene therapy to improve the vision of both eyes in three women who were born virtually blind.

The new proceeding has extended earlier work by Jean Bennett and colleagues who used gene therapy to treat blindness in 12 adults and children with Leber's congenital amaurosis (LCA).

The rare inherited eye condition affects vision by destroying the light sensitive cells in the retina at the back of the eye.

The new trial was carried out on three patients who were successfully treated in one eye by the same team years ago.

After using the experimental therapy for their second eyes, their vision has significantly improved and now they can avoid obstacles and distinguish faces even in dim light, says the report published in the journal Science Translational Medicine.

“After the treatment, their vision is by no means 20/20, but it’s improved to the point where they can see the faces of their children, see the teacher at a school conference, or see their child hit a home run in a baseball game,”‌ said Bennett.

The findings not only reconfirmed the effectiveness of using gene therapy for patients with LCA but also removed much of the scientists’ concerns about its possible side effects including immune system reactions.

The novel treatment doesn’t make patients’ vision perfect but extensively improves their quality of life. “They can’t read books or drive a car,”‌ Bennett said. “But the improvements mean a huge amount to somebody who is blind.”‌

Source: presstv.com