Gene Therapy to Correct a Common Form of Blindness

University of Florida (UF) researchers have discovered a new technique called gene therapy to replace a malfunctioning gene in the eye with a normal copy that supplies a protein necessary for light-sensitive cells in the eye to function. The research study co-authored by William W. Hauswirth opined that, "Providing the gene that's missing is one of the ultimate ways of treating disease and restoring significant visual function."

The disease, retinis pigmentosa is a genetic defect carried by the female genome but vision loss is manifested in boys by initial loss of peripheral and night vision, which eventually leads to blindness.

Collaborative efforts between UF and the University of Pennsylvania led to the cloning of the gene into a viral vector that served as a vehicle to transport it to the appropriate part of the eye. A genetic switch that would turn on the gene was included so that protein production can commence once the gene is in place. Laboratory tests involving diseased animals was successful and plans to repeat the studies on a larger scale and make a version of the virus safe for humans is in the offing.

The news story can be viewed at http://news.ufl.edu/2012/01/23/cure-blindness/