Young Man’s Sight Restored by Gene Therapy

A team of specialists at London’s Moorfields Eye Hospital in Britain have restored a young man’s sight by using gene therapy. Eye specialists reported that the gene therapy regenerated the dying eye cells in the third of such operations. While the gene therapy has not improved the vision of the other two patients it may well stop their vision from declining further. This success has wide implications as it establishes that gene therapy can work and suffering people can be alleviated. The positive results could also lead to treatments for several other conditions, including blood and immune disorders.

The man, 17-year-old Stephen Howarth suffers from Leber’s Congenital Amaurosis which is caused by a fault in a single gene, and leads to a progressive loss of sight. Within a few months, doctors noted some improvements but Stephen himself did not realize these changes until he confidently walked through a dimly-lit maze designed to test his vision and surprised his doctors with the level of his improvement. Without the operation it was likely that Stephen would have lost his sight altogether.

The research is published in the current edition of the New England Journal of Medicine.