For the first time, researchers have used gene therapy to increase light sensitivity and improve vision in patients who were virtually blind, a finding that offers new hope to hundreds of thousands of patients with inherited forms of vision impairment.Researchers used the treatment for a type of inherited blindness and said they found it to cause no side effects, and slight improvements in vision in four out of six patients studied The scientists used a vector, a genetically engineered adeno-associated virus, to carry a normal version of the gene, called RPE65, that is mutated in one form of LCA. Three patients, ages 19, 26 and 26, received the gene therapy via a surgical procedure performed by Maguire between October 2007 and January 2008 at The Children's Hospital of Philadelphia, where the gene vector was manufactured at the hospital's Center for Cellular and Molecular Therapeutics (CCMT). Starting two weeks after the injections, all three patients reported improved vision in the injected eye. The LCA gene therapy vector showed no signs of causing inflammation in the retina or other toxic side effects.
Gene transfer has been in clinical trials for over 15 years now, and although it has an excellent safety record, examples of therapeutic effect are still relatively few. The results in this study provide objective evidence of improvement in the ability to perceive light, and thus lay the groundwork for future studies in this and other retinal disorders,
In a nutshell, gene therapy is a relatively new class of experimental treatments aimed at replacing or fixing defective genes. Past gene therapy clinical trials have met with limited success despite high hopes. This trial establishes proof of principle of gene therapy for inherited retinal disease and paves the way for the development of gene therapy approaches for a broad range of eye disorders .
Fong Jiuan
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