Tags: gene | therapy | blindness

Gene Therapy Treats Blindness

Thursday, 09 Feb 2012 01:34 PM



Doctors have successfully used a new gene therapy technique to restore sight to three patients with congenital blindness
Scientists from Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania led the study, published in the journal Science Translational Medicine.
The three patients have a rare inherited disease -- Leber's Congenital Amaurosis (LCA) -- caused by genetic defects. It typically appears at birth or in the first months of life, leading to severely impaired vision.
The new treatment involves boosting a faulty gene that causes the condition by injecting one eye with an engineered virus carrying the gene RPE65. In the new study, researchers found treating both eyes of all three patients improved their vision significantly.
"Patients have told us how their lives have changed since receiving gene therapy," said study co-leader Dr. Jean Bennett, in statement. "They are able to walk around at night, go shopping for groceries and recognize people's faces -- all things they couldn't do before. At the same time, we were able to objectively measure improvements in light sensitivity, side vision and other visual functions."
The researchers said follow-up studies must be done with additional patients. But Bennett said the research holds promise for using a similar gene therapy approach for other eye diseases.

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Blind patients treated with a new gene therapy technique have regained their sight, scientists report.
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2012-34-09
Thursday, 09 Feb 2012 01:34 PM
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