Gene Therapy Success Restores Vision

This is exciting news and demonstrates once again that most biotechnology isn’t morally contentious. A teenager with failing eyesight has had his vision improved by inserting healthy genes to correct a genetically caused disability. From the story:

In the trial carried out by a team at the University College of London Institute of Ophthalmology and Moorfields Eye Hospital, the world’s first gene transplant for blindness produced an unprecedented improvement in Steven Howarth’s sight.

The student suffered from a genetic mutation, called Leber’s congenital amaurosis (LCA), which usually begins affecting the sight of sufferers in early childhood and eventually causes total blindness during a patient’s twenties or thirties. Currently, there is no treatment for the condition.

Born with no peripheral or night vision, he noticed a marked improvement after the two-hour operation.”Now, my sight when it’s getting dark or it’s badly lit is definitely better,” he says. “It’s a small change—but it makes a big difference.”

Prof Robin Ali, the head of the team, said the evidence of his improvement was “compelling”. The doctors injected genes only into Mr Howarth’s worst affected eye and used the lowest dose in what was strictly a safety trial.

And that’s an important point. Gene therapy can be dangerous, as Jesse Gelsinger tragically found out several years ago: It killed him.

But this is very hopeful news and a good sign for the future of regenerative medicine.