Blind Gene Therapy
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A LIGHT AT THE END OF THE TUNNEL MAY SOON APPEAR FOR THOSE SUFFERING A FORM OF GENETIC BLINDNESS. UNIVERSITY OF FLORIDA RESEARCHERS HAVE DISCOVERED THAT RETINAL GENE THERAPY CAN IMPROVE THE EYESIGHT OF PEOPLE BORN WITH A GENE DEFECT THAT ROBS THEM OF THEIR VISION.
Dr. William Hauswirth/UF researcher: “The gene essentially goes into the cells that is missing it, and it starts expressing the protein that it encodes, and that protein is the enzyme they’re missing. So, this enzyme then restores the visual cycle to these cells.”
UF RESEARCHERS INJECTED THE GENE INTO THE EYES OF THREE YOUNG ADULTS WITH A TYPE OF BLINDNESS CALLED LCA2. THEY FOUND THE PATIENTS EYES WERE TEN THOUSAND TIMES MORE FUNCTIONAL IN THE REGION THE THERAPY WAS INJECTED INTO. ONE WOMAN COULD SEE A DIGITAL CLOCK FOR THE FIRST TIME IN HER LIFE.
Dr. William Hauswirth/UF researcher: “Her brain had realized that we had restored some function to a non-central part of her retina, and her brain had now re-wired the muscles of her eye so that depending on the brightness of the object, she would use either the central retina or the treated part of the retina.”
PEOPLE WITH SIGHT HAVE ONE CENTRAL REGION, OR FOVEA, THEY CAN SEE OUT OF. BUT, THIS GENE THERAPY CREATED ANOTHER OFF-CENTER REGION THAT HAS BECOME MORE SENSITIVE TO LIGHT.