Researchers from Columbia University have developed a new technique for the powerful gene editing tool CRISPR to restore retinal function in mice afflicted by the degenerative retinal disease, retinitis pigmentosa. As this is the first time the CRISPR technology has been shown to work on an inherited disease the same tool might work in hundreds of diseases, including Huntington’s, Marfan syndrome and corneal dystrophies, said researchers in Ophthalmology.
Dr Stephen Tsang and his colleagues calls the technique genome surgery because it cuts out the bad gene and replaces it with a normal, functioning gene. He expects human trials to begin in three years.
“Genome surgery is coming,” said Dr Tsang. “Ophthalmology will be the first to see genome surgery before the rest of medicine.”