MONDAY, April 28, (News Locale) - That gene therapy is now the future of medicine was underlined by a case report wherein doctors were able to regenerate dying cells in the eye of a youth who had inherited a rare type of blindness. 

Two new studies published in the New England Journal of Medicine say that gene therapy may be the answer to a rare disease called Leber's congenital amaurosis (LCA). The condition is inherited at birth and signs of visual problems begin to appear shortly after birth.

In the reported studies, three Italian patients and one patient from London were able to see after gene therapy. BBC News reports that the London patient who received gene therapy is a 17-year-old named Stephen Howarth.

Prior to gene therapy, Howrath had nearly los his vision and used to routinely walk into walls. However a team led by Professor Robin Ali, of the Institute for Ophthalmology say that the therapy has proved to be far more successful than they imagined. Not only has Stephen's vision improved, he can also hope to reverse the condition altogether.

Leber's congenital amaurosis (LCA) arises because of a glitch in the RPE65 gene. The condition results in degeneration of the light receptor cells in the retina located at the back of the eye. LCA is a progressive disease and nearly all affected patients go blind by the age of 30. There is no known cure for the disease.

LCA starts off as nystagmus (involuntary eye movement), sluggish or no pupillary responses, and eventually leads to severe vision loss or blindness.

Researchers at Moorfields Eye Hospital and University College London in London carried out the first gene therapy on LCA in a patient named Robert Johnson in 2007. The operation was carried out by using a synthetic virus to obtain healthy copies of the defective gene. After the healthy genes were obtained, the mixture was injected deep into the retina of the patient.

The virus used by the researchers was made by Seattle-based made by Targeted Genetics Corp.

In the second study, researchers used a virus made at the Children's Hospital of Philadelphia. Researchers report that patients in this study were able to see dim areas more clearly two weeks after the gene therapy surgery.

Both studies were also presented at the Association for Research in Vision and Ophthalmology meeting in Ft. Lauderdale, Florida.

Gene therapy promises to be the elixir that will cure all genetically inherited diseases. While the above studies have reported initial success, researchers have stressed the need for bigger and longer studies to accurately gauge the results of genetic intervention.

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