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Gene therapy used to try and cure type of blindness

Published on May 2, 2007 at 2:59 AM · No Comments

A team of doctors from Moorfields Eye Hospital and University College London (UCL) have carried out world's first eye operations using gene therapy to try to cure a serious eye disorder.

The patient Robert Johnson was born with Leber's congenital amaurosis (LCA), a type of inherited childhood blindness caused by a single abnormal gene; having the disorder means he can see very little at night, and he can expect his sight to deteriorate even further with age.

The team say it will be several months before the researchers know whether their work with Mr. Johnson and 11 other has been a success but the doctors say there have been no complications so far.

LCA is an inherited degeneration of the retina and the condition prevents the retina from detecting light properly, resulting in progressive deterioration and severely impaired eyesight. There is currently no effective treatment.

In this form of retinal degeneration the photoreceptor cells are present, but not functioning.

The aim is to restore the activity in the photoreceptor cells and therefore restore vision by implanting healthy copies of the key gene into the RPE65 gene into cells of the retina using a harmless virus or vector.

The team of doctors are collaborating with Seattle, Washington-based biotech firm Targeted Genetics which made the vector being used in the trial.

Testing the procedure on humans follows 15 years of laboratory and animal experimentation, including tests on dogs whose vision was restored to the extent they could navigate a maze with ease.

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