Gene therapy study shows promise for Retinitis pigmentosa

A study published in the April 2010 issue of the Federation of American Societies for Experimental Biology (FASEB) Journal details the results of using nucleic acid nanoparticles to restore the vision in mice. This form of gene therapy does not involve the use of modified viruses. Scientists from Buffalo, Cleveland and Oklahoma City, used a non-viral, synthetic nanoparticle to restore the vision of mice with retinitis pigmentosa. Retinitis pigmentosa is an inherited disease which causes cells in the retina to die prematurely. This results in progressive vision loss and ultimately, irreversible blindness. The mice in the study which received the nanoparticle gene therapy showed considerable signs of healing as well as structural improvement in their retinas and functional vision improvements. All of these effects lasted throughout the course of the study. In addition, no adverse side effects were noted. This is very encouraging research for a disease that up until now has had no hope for stabilization of eyesight or cure.* Andrea Schumann Staff Writer