Scientists in China have successfully used the revolutionary gene-editing tool Crispr to restore vision in blind mice, according to research published Friday in the Journal of Experimental Medicine. The mice had a genetic condition, retinitis pigmentosa, which causes cells in the retina to break down and is one of the major causes of blindness worldwide.
The scientists used a new Crispr tool to repair the mutation responsible and found that the mice kept their vision even into old age. The researchers assessed their visual skills using behavioral tests and found they could, for example, escape from an aquatic maze.
Crispr technology has been used to restore vision to blind mice before, but the tools used were not capable of efficiently correcting the genetic causes of blindness in humans, including retinitis pigmentosa. The new tool developed by the scientists is able to “carry out any type of editing at any location in the genome” and provides “much more convincing evidence” for the diverse array of possible uses gene editing might have for treating disease.
Encouraged by the results, the researchers are now keen to test the tool against other types of retinitis pigmentosa, which can be triggered by mutations in over 100 different genes. However, human applications are still a long way off, as “much work still needs to be done to address safety concerns and ensure efficacy before this system can be used safely and effectively in humans.”
In the US, an estimated 1 in 4,000 people are affected by retinitis pigmentosa, making this breakthrough an exciting step forward in the development of gene editing technology and its potential applications in humans.