Cell: Recover vision with the help of genetic marker technology CRISPR
Using the genetic marker technology "CRISPR/CAS9 " , researchers from the University of California, San Diego, and researchers from China were able to repair the mutant rod-shaped optical receptors and restore their function to normal. This technique succeeded in making two different The vision of mice with a type of retinal degenerative disease returned to normal. The results were published in the recent issue of Cell Research.
Retinitis pigmentosa (RP) is a type of hereditary visual impairment disease in which more than 60 genes are mutated. These mutant genes affect the photoreceptors of the eye (ie, a type of sensory light in the eye that converts it into a special cell type that is transmitted to the brain by electrical signals). There are currently two kinds of photoreceptor cells. The rod cells are mainly responsible for the nighttime light signals and the residual light signals. The cone cells are responsible for the central visual signals and color resolution. People's retina contains 120 million rod cells and 6 million cone cells.
Worldwide, one in every 4,000 people has RP. This rod-specific gene mutation causes the function of the rod-shaped photoreceptor to be inactivated, and the function of the cone-shaped cells is also disordered. There is currently no effective treatment plan for RP.
In this study, the authors used the CRISPR/CAS9 system to inhibit the activity of a key gene, Nrl, and the downstream transcription factor Nr2e3. This method is capable of differentiating rod-shaped cells into cone-shaped cells. "The genetic mutations in the cones trigger a relatively low risk of RP, and our protocol is therefore able to reduce the incidence of RP symptoms. "
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