Scientists in Shanghai have successfully turned glial cells in the brains of experimental mice into neurons by gene editing, providing a new way to further treat neurodegenerative diseases.

In this study, the researchers by the application of the latest gene editing tools, RNA targeting CasRx CRISPR system, on the mice retina muller glial cells and astrocytes in brain striatum gene editing, induced the transdifferentiation of optic ganglion cells and dopamine neurons, which proves that these two by glial cells "transformation" of neurons, the mice can be restored by optic ganglion cells death and permanent damage eyesight, eliminate die in dopamine neurons in mice and the symptoms of Parkinson's disease.

Optic ganglion cells are the only bridge between the eye and the brain. Acute ischemic retinopathy, chronic glaucoma and other eye diseases can lead to the death of optic ganglion cells. According to statistics, glaucoma alone causes blindness in the world more than 10 million people. Parkinson's disease, which is caused by the death of dopamine neurons in the substantia nigra region of the brain, currently affects nearly 10 million people worldwide, about half of whom are in China.

In this study, the researchers first achieved the regeneration of optic ganglion cells in adult mice by specifically knocking down the expression of Ptbp1 gene in mouse retinal muller glia, and the regeneration of optic ganglion cells restored permanently damaged vision in mice. Then show that the strategy can also be specific to the striatum of the brain astrocytes and efficient by the turn of the dopamine neurons, and these transdifferentiation of dopamine neurons can make up for the lack of the function of the dopamine neurons, which will reverse movement disorder Parkinson's disease model mice to levels close to normal mice.

Previous studies have focused on directly reducing mutations in harmful genes that cause disease, but this study is a therapeutic "fate" transformation of cells in the body, providing a new perspective for disease research and drug development.

Source:http://www.cebsit.cas.cn

Time: 2020.04.09