Washington, Oct 26 (Inditop.com) Children with congenital blindness may be able to improve their condition with gene therapy, say researchers.
“Children who were treated with gene therapy are now able to walk and play just like any normally sighted child,” said study co-author Albert M. Maguire, associate professor of ophthalmology at Pennsylvania State University (PSU) and physician at the Children’s Hospital in Philadelphia.
The study, conducted by PSU researchers and the Centre for Cellular and Molecular Therapeutics at The Children’s Hospital, used gene therapy to safely improve vision in children and adults with Leber’s congenital amaurosis (LCA).
The greatest improvements occurred in children, all of whom are now able to navigate a low-light obstacle course — one result that the researchers call “spectacular”.
“This result is an exciting one for the entire field of gene therapy,” said Katherine A. High, study co-author.
“This study reports dramatic results in restoring vision to patients who previously had no options for treatment,” said High. “These findings may expedite development of gene therapy for more common retinal diseases, such as age-related macular degeneration.”
The study appeared in the Sunday online edition of The Lancet.
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