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Eye Gene Therapy

new hope for the blind London,

Diagram of the inside on the eye.
Improved vision following gene delivery

Thousands of people with failing eyesight have been given fresh hope of a treatment after gene therapy techniques have been successfully used.

In 2008 a team at University College London's Institute of Ophthalmology and Moorfields Eye Hospital led by Robin Ali reported the world's first gene transplant for blindness, which produced an unprecedented improvement in the sight of patient Steven Howarth.

The treatment, in which healthy genes were injected into his worst affected eye, transformed the life of the severely visually impaired 18-year-old student from Bolton who suffered from a genetic mutation that caused a degenerative eye condition called Leber's congenital amaurosis (LCA).

The technique has wider uses in treating 100 or so  inherited diseases of the retina, which affect 20,000 people in Britain. This type of therapy is now being tested on people with other types of inherited retinal conditions and on those  who suffer age related macular degeneration,  the most common cause of blindness.

Gene therapy will be useful for patients who still have remaining retinal cells, but Ali is also developing stem cell therapy to replace cells that have already been lost.

Royal Society

Biology, Medicine,
University College London Institute of Ophthalmology; Moorfields Eye Hospital
Key Individuals
Robin Ali,