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Gene therapy offers hope for LCA patients

Clinical
US scientists have claimed a success in using gene therapy to treat patients with the inherited condition Leber's congenital amaurosis (LCA).

US scientists have claimed a success in using gene therapy to treat patients with the inherited condition Leber's congenital amaurosis (LCA).

According to a report published on BBC Online, researchers from Pennsylvania University, the University of Florida and Cornell University, injected material using a corrective gene into the eyes of three patients with the condition.

Researchers say all three patients showed signs of 'significant' improvement in their vision, according to findings published in the Journal Proceedings of the National Academy of Sciences.

They also claim that within three years they could be ready to carry out a similar procedure on patients with age-related macular diseases.

LCA affects around 80,000 people in the UK and the condition is responsible for one in 10 severe sight disorders in children.

This is not the first time researchers have used gene therapy to treat LCA patients.

As reported in Optician earlier this year, (News, May 2), researchers from the UCL Institute of Ophthalmology and Moorfields Eye Hospital NIHR Biomedical Research Centre announced results from what they claimed was the world's first clinical trial of gene therapy to treat patients with the condition.

Three patients were treated as part of their clinical trial, which started in February 2007, and resulted in one of the patients benefiting from significantly improved night vision.

At the time researchers claimed that this was demonstrated by the patient's ability to negotiate a simulation of a night-time scene quickly and without mistakes, something he had been unable to do prior to the surgery.




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