Researchers from Columbia University have successfully applied CRISPR gene editing technology to restore retinal function in a mouse model. Their study was recently published online in Ophthalmology.
The mice were afflicted with retinitis pigmentosa, a rare inherited disorder that causes breakdown and loss of cells in the retina. Affecting roughly 1 in 4,000 people worldwide, it typically strikes in childhood and progresses slowly. Most will lose much of their sight by early adulthood and become legally blind by age 40.
The team used a modified CRISPR technique which added two guide RNAs instead of one. The addition improved the chance of correcting a deleterious variant of the rhodopsin gene from 30 to 90 percent. The components of the CRISPR machinery were delivered into the retina using an adeno-associated virus.
Another advantage is that this technique can be used in non-dividing cells, which means that it could enable gene therapies that focus on nondividing adult cells, such as cells of the eye, brain, or heart. Up until now, CRISPR has been applied more efficiently in dividing cells than non-dividing cells. An electroretinogram evaluated the mice after treatment, showing a significant improvement in retinal function.
“Genome surgery is coming. Ophthalmology will be the first to see genome surgery before the rest of medicine,” said Stephen H. Tsang, senior author of the study.
See the full news release from American Academy of Ophthalmology.
Source: American Academy of Ophthalmology