【Animal Experiment】-Using CRISPR/Cas9 system to rescue blind mice
动物实验,CRISPR/Cas9系统
z-bo
2020-08-28
454
The method of gene editing can prevent the degeneration of the mouse retina, thereby saving blind mice. The described method uses the CRISPR/Cas9 gene therapy system and can be applied to a variety of potential genetic defects that cause retinitis pigmentosa (the main cause of blindness). Retinitis pigmentosa is an incurable disease that is difficult to detect and severely damages the eyes and genetics of patients. Although the disease is characterized by retinal degeneration, retinitis pigmentosa may be caused by mutations in more than 60 genes, so it is difficult to develop targeted therapies to repair each specific gene. Yes. The retina contains rods and cones. The genetic mutation that causes retinitis pigmentosa first leads to rod death, then cone death, and finally blindness. I will.
Wu Zhijian, a researcher at the National Institute of Ophthalmology (transliteration) in the United States, and his colleagues did not cure the disease that caused the gene mutation this time. Instead, they tested a method for preserving pyramidal cells. They use the CRISPR/Cas9 system, which is the most popular "star" technology in the biomedical field, to invalidate the genes that determine the rod's identity and ensure that the rod acquires the characteristics of cone cells. Inducing protection from harmful diseases. The impact of mutations. Experimental results show that in three mouse models of retinal degeneration (30 mice in total), the therapy can prevent retinal degeneration and improve vision. Studies have shown that this method may be suitable for the treatment of retinal degenerative diseases, and may not be affected by a wide range of potential genetic mutations and has broad applicability. Being displayed. in
Chief Editor
In the past, gene therapy usually used viruses to introduce and replace target genes. The advantage of CRISPR is that it does not introduce foreign genes and avoids related risks. However, providing CRISPR to the target location to make it effective remains a challenging technical challenge. With the latest progress in mice, more optimism. I am not sure whether CRISPR will become the main tool for gene therapy in the future, but if not, then there are suitable genetic tools, which are as simple as appendicitis surgery, and can now completely cure incompetent genetic diseases.
