According to a recent article that was published in The Independent, a young man by the name of Steven Howarth can now see again thanks to gene therapy. Howarth, an 18 year-old from Bolton in the UK, had a rare eye disorder that left him with extremely poor vision as well as the inability to see in the dark under low light conditions. The disorder, Leber’s congenital amaurosis, is an inherited degenerative disorder that leads to progressive loss of sight and is caused by a faulty gene (RPE65). However, due to an injection that was given to Howarth’s affected eye, his vision has improved significantly.

Robin Ali is a professor of human molecular genetics at UCL Institute of Ophthalmology and led the British study. The procedure involved administering the missing gene with a single injection to the back of the retina of the affected eye. In Howarth’s case, the affected eye had almost no vision, especially in low light such as street lights at night. Howarth also demonstrated extreme difficulty in navigating through a maze of three doorways, repeatedly bumping into walls and losing his sense of direction. However, six months after receiving the gene therapy treatment, Howarth was, again, put into a maze with low light and surprised doctors and researchers by navigating the maze with no difficulty at all. The results of the study were published in the New England Journal of Medicine.

Professor Ali was pleased with the treatment and was cited as saying:

This is a very significant milestone. This trial establishes proof of principle of gene therapy for inherited retinal disease and paves the way for development of gene therapy approaches in a broad range of eye disorders.

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