Gene Breakthrough Aids Blind

Experimental therapy improves sight
By Peter Fearon,  Newser Staff
Posted Apr 28, 2008 2:42 AM CDT
Gene Breakthrough Aids Blind
A patient undergoing a PET scan after gene therapy at the Hospital of the University of Pennsylvania in Philadelphia. Doctors at the hospital report a breakthrough impacting the whole field.   (KRT Photos)

A rare and incurable congenital disease which causes blindness has been successfully treated for the first time using gene therapy, Reuters reports. Doctors inserted a healthy gene directly into one eye of patients suffering from Leber congenital amaurosis using a genetically engineered cold virus. Patients reported an improvement in their eyesight even though none was expected.  

The disease damages light receptors in the retina and usually causes total blindness by the age of 30. "This result is important for the entire field of gene therapy," said the lead researcher of one of the studies published in the New England Journal of Medicine. Genetic research to treat illness suffered serious setbacks in recent years when some experiments went awry. (More gene therapy stories.)

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