New Gene Therapy Could Cure Eye Disorders


New research suggests that gene therapy could cure eye disorders that lead to blindness. The research, published in the April edition of the Journal of American Association of Paediatric Ophthalmology and Strabismus, seems to show that a particular form of gene therapy could be used to restore sight in people who suffer from Leber congenital amaurosis (LCA). LCA is a congenital retinal disorder that is thought to affect one in every 80,000 people. It is hoped that the research will lead to the development of improved treatment methods that will ultimately restore sight to those who have become blind because of inherited and genetic eye disorders.

The study, conducted by researchers from the University of Iowa and Toronto’s Hospital for Sick Children, found that LCA and other related eye degenerative disorders can be caused because of genetic mutation across a number of genes. However, the researchers found that LCA caused by a mutation in the RPE65 gene could be treated by using a ‘normal’ copy of the gene. The team now hope to use genetic screening to identify and treat patients who will be able to successfully respond to treatment.

This new gene therapy is currently undergoing clinical trials and researchers hope that their findings can be used to effectively treat people who have impaired sight or lost their sight due to LCA. They also hope to further research LCA in order to find similar gene therapy treatments that can restore sight in individuals who have one or more of the 14 other genetic mutations that can be responsible for causing the disorder.

7th April 2010

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