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Ohio State researchers design a viral vector to treat a genetic form of blindness
Date:11/2/2011

COLUMBUS, Ohio Researchers at Ohio State University Medical Center and Nationwide Children's Hospital have developed a viral vector designed to deliver a gene into the eyes of people born with an inherited, progressive form of blindness that affects mainly males.

The vector is part of a clinical trial investigating the use of gene therapy to cure choroideremia, a disease that affects an estimated 100,000 people worldwide. The trial is being conducted by researchers at the University of Oxford in England.

The vector was designed by Dr. Matthew During, professor of molecular virology, immunology and medical genetics and of neuroscience and neurological surgery at Ohio State, in collaboration with Robert MacLaren, professor of ophthalmology at the University of Oxford, who also leads the trial.

Researcher Dr. K. Reed Clark, director of the Clinical Manufacturing Facility at the Center for Gene Therapy, Nationwide Children's Hospital, and his team produced the clinical-grade vector that is administered to patients in the trial.

During, who is also a visiting professor of translational neuroscience at Oxford, was in the operating room during the pioneering surgery. "I and my colleagues are excited about contributing to this significant medical breakthrough," During says. "We have worked for many years to engineer and optimize viruses to safely deliver genes to humans, and the eye is an ideal target in many ways. The clinical vector manufacturing facility at Nationwide Children's Hospital is outstanding, and Dr. Clark and his team deserve congratulations for providing a clinical vector that for the first time offers these patients the possibility of an effective therapy."

During and his colleagues designed the viral vector to infect the light-sensitive photoreceptor cells that line the back of the eye and make up the retina. Choroideremia causes a degeneration of these photosensitive retinal cells and progressive blindness
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Contact: Darrell E. Ward
darrell.ward@osumc.edu
614-293-3737
Ohio State University Medical Center
Source:Eurekalert

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