编辑干细胞疗法,失明 z-bo 2020-10-20 380

　　In the latest research paper published in the international journal "Science Reports", researchers at Columbia University and others used CRISPR/Cas9 gene editing technology to successfully correct genetic mutations that caused blindness in patients' stem cells. Related research results may be useful for future development. New therapies for personalized treatment of eye diseases provide new clues. Researcher Alexander Bassuk said that our goal is to repair the degenerative retina of X-chain retinitis pigmentosa (XLRP) patients. CRISPR/Cas9 is a precise gene editing technology that can effectively correct damaged RPGR. A single DNA change in the gene, and more importantly, this modified tissue is derived from the patient’s own stem cells and can be transplanted into the patient without the use of harmful drugs to inhibit tissue rejection.

　　By applying CRISPR/Cas9 technology to human stem cells, researchers can correct specific genetic mutations before transplanting new healthy cells into patients. Retinal disease is an ideal model for the development of stem cell therapy. Advanced surgical techniques can be used to inject cells to the desired location. The research in this article is a verification experiment. Related results show that researchers can not only repair rare genetic mutations, but also manipulate stem cells in patients. The use of stem cells is key because stem cells can be reprogrammed into retinal cells.

　　Currently, CRISPR technology can correct 13% of RPGR mutations in stem cells. Researcher Bassuk pointed out that the results of this study are promising, because gene mutations are usually located in very repetitive sequences of the RPGR gene and actually identify specific DNA sequences in the gene. This is very difficult, and researchers don't know whether CRISPR/Cas9 technology can correct mutations at gene points. CRISPR technology has not been used in human research before. Many scientists worry that the technology will cause potential genetic mutations in the body, while the Ethical Society believes that the technology will change human sperm and egg cells, which researchers are still doing. There is a lot to do. They hope to monitor and study patients with special retinal degeneration diseases in real time to help develop new disease treatment methods.