Gene Therapy

Gene Therapy Provides Vision to People who Were Nearly Blind

Published in Gene Therapy Weekly, May 15th, 2008

Scientists employing a gene therapy have provided partial vision to patients who were nearly blind from a condition known as Leber congenital amaurosis (LCA) -- a severe form of retinitis pigmentosa. Initial results from the clinical trial, which was funded in part by the Foundation Fighting Blindness, were published in the New England Journal of Medicine.

All three patients, who had severely abnormal vision before entering the study, can now read several lines on an eye chart and are able to see better in dimly lit settings. One was also able to navigate better after the injection.

"This breakthrough is the greatest advancement in the 37-year...

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Source: Gene Therapy Weekly (2008-05-15)

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