Gene Therapy Treatment Successful For Severe Hereditary Eye Disease

October 25, 2009

(ChattahBox) — A team of doctors in the U.S. treated 12 patients who have a rare genetically inherited retinal degeneration called Leber congenital amaurosis (LCA), by using gene therapy which significantly improved vision. People with LCA, which is caused by mutations in any of least 13 genes, have severe vision loss and abnormal eye movements in early infancy and during early childhood. Most people with the disease are blind by their 30s or 40s.

The new phase I trial included 12 patients, aged 8 to 44 years, who were injected in one eye with genetic material on a virus, meant to correct LCA. The gene therapy led to at least a 100-fold increase in pupillary light response (constriction of the pupil when it’s exposed to light) in the participants. Younger patients seemed to react better to the gene therapy, with  8-year-old patient developed nearly the same level of light sensitivity as a person with normal vision, the study authors reported. Six of the 12 patients had so much of their sight restored, that they may no longer be classified as legally blind.

“All 12 patients given gene therapy in one eye showed improvement in retinal function. The effect was stable during follow-up. The results support our hypothesis that the response to subretinal gene therapy depends on the extent of retinal degeneration and, therefore, the age of the patient,” wrote Dr. Jean Bennett, of the University of Pennsylvania School of Medicine in Philadelphia, and colleagues.

The study is published in the Oct. 24 online edition and in an upcoming print issue of The Lancet.

Source: BreakThroughDigest Medical News


One Response to “Gene Therapy Treatment Successful For Severe Hereditary Eye Disease”

  1. Wellescent Health Blog on October 25th, 2009 10:25 am

    This finding is impressive both for those who suffer the condition itself and for the the improvements in the science that are allowing the use of viruses in repairing genetic defects in such a precise manner. It would be interesting to know the reason why a younger age leads to better success in the treatment.

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