New treatment for inherited blindness allows people to see well enough navigate a maze

The treatment could pave the way for treatment of other inherited retinal diseases and more common loss of vision due to age degeneration (Unsplash/Vanessa Bumbeers)
Ella Wills11 November 2017

A new genetic treatment for inherited blindness may soon be available, researchers have suggested.

Patients who lost their sight to an inherited retinal disease could see well enough to walk around a maze after a new gene therapy, according to new research.

The study showed how a breakthrough gene therapy improved vision in 93 per cent of patients with a condition called Leber Congenital Amaurosis (LCA), which begins in childhood and eventually causes complete blindness.

The therapy is under review by the US Food and Drug Administration (FDA) for potential approval this year.

There are currently no treatments available for inherited retinal diseases.

In the study, 93 per cent of 29 treated patients experienced meaningful improvements in their vision.

This was enough that the patients could navigate a maze in low to moderate light levels.

They also demonstrated improvement in light sensitivity and peripheral vision, which are two visual deficits these patients experience.

Led by Stephen R Russell from the University of Iowa, the research could open the door for other gene therapies to treat more than 225 genetic mutations known to cause blindness.

Researchers also suggested the gene therapy could one day help to restore vision in more common diseases, such as age-related macular degeneration – a leading cause of vision loss for people over the age of 50.

LCA is a rare condition, affecting around 1 in 80,000 individuals, researchers said.

It can be caused by one or more of 19 different genes.

The treatment, called voretigene neparvovec (Luxturna, Spark Therapeutics), involves a genetically modified version of a harmless virus. The virus is modified to carry a healthy version of the gene into the retina.

Doctors inject billions of modified viruses into both of a patient's eyes.

While treatment does not restore normal vision, it allows patients to see shapes and light. They are then able to get around without a cane or guide dog.

Most patients in the study have maintained their vision for two years, but it is unclear how long the treatment will last.

An advisory committee for the FDA endorsed the treatment for approval in October. The FDA is expected to make its decision by January 2018.

The research was presented at an annual meeting of the American Academy of Ophthalmology on Friday.

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