Using gene editing, scientists from the Wuhan University of Science and Technology have restored the vision of mice that lost their sight due to retinitis pigmentosa. Research published Journal of Experimental Medicine.
Retinitis pigmentosa occurs in 1 in 4,000 people and can be caused by mutations in more than 100 genes. Some patients experience moderate visual impairment, while others may lose it completely.
In the new study, the biologists focused on a mutation in the gene encoding the PDE6B enzyme, which can cause complete vision loss. They developed a universal PESpRY gene editing system based on the CRISPR system to edit it. It can be used to correct many types of genetic mutations, wherever they occur in the genome.
Editing the gene prevented the death of rods and cones in the retinas of the mice and restored their normal response to light. The animals can navigate the maze almost as well as normal healthy mice. They exhibited typical head movements in response to visual stimuli. The eyesight of mice was preserved until old age.
If the new gene editing method proves safe and effective, it could be used to treat humans.