Gene therapy safety trial for childhood blindness...( GAINESVILLE Fla. Three decades have...),Health

GAINESVILLE, Fla. Three decades have passed since gene therapy pioneer William W. Hauswirth, Ph.D., and his colleagues at the University of Florida began work on a virus that could safely deliver corrective genes into living animals.

Its been six years since a multi-university team used gene therapy to give sight to puppies born with a defect that causes blindness.

Now the gene-transfer technique is being tested for safety in people in a phase 1 clinical research study conducted by the University of Pennsylvania and the University of Florida with support from the National Eye Institute of the National Institutes of Health.

A young adult with a form of hereditary blindness called Leber congenital amaurosis type 2, or LCA2, received an injection of trillions of replacement genes into the retina of one eye this month, making the volunteer one of the first people in the world to undergo the procedure. Shalesh Kaushal, M.D., Ph.D., an assistant professor of ophthalmology at UF, performed the gene transfer.

The volunteer was discharged last week from the General Clinical Research Center at UF.

In all, six adults and then three children between the ages of 8 and 17 will undergo the gene-transfer procedure at UF over the next year or more before safety data are fully evaluated. Names are not being disclosed for privacy reasons. Potential risks are discussed with prospective participants as part of an extensive screening and informed consent process.

This is the first study of its kind to investigate inherited blindness, said Barry J. Byrne, M.D., Ph.D., a professor of molecular genetics and microbiology and director of UFs Powell Gene Therapy Center. The accomplishment reflects a great deal of work and dedication on the part of Dr. Hauswirth, as well as many other scientists and physicians, including Samuel G. Jacobson, M.D. Ph.D., professor of ophthalmology of the University of Pennsylvania, and literally dozens
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