编辑干细胞疗法 z-bo 2020-12-16 455

　　Recently, in a research paper published in the international journal Scientific Reports, researchers from Columbia University and others have used CRISPR/Cas9 gene editing technology to successfully correct the blindness-causing genetic mutations in patients’ stem cells. The relevant research results may be later. The development of new therapies for individualized treatment of eye diseases provides new clues.

　　Researcher Alexander Bassuk said that our goal is to repair the degenerated retina in the eyes of patients with X-linked retinitis pigmentosa (XLRP) disease. CRISPR/Cas9 is a precise gene editing technology that can effectively correct the damage. A single DNA change in the RPGR gene, and more importantly, this modified tissue is derived from the patient’s own stem cells, which may be able to be transplanted to the patient without using harmful drugs to suppress tissue rejection. With the application of CRISPR/Cas9 technology in human stem cells, researchers can transplant new healthy cells to patients after correcting specific genetic mutations. Retinal diseases are the perfect model for stem cell therapy development because researchers can use Advanced surgical techniques to inject cells into their desired location. The study in this article is a verification experiment. The relevant results show that researchers can not only repair a rare genetic mutation, but also manipulate stem cells in the patient's body. The use of stem cells is the key because they can be reprogrammed into retinal cells. . At present, CRISPR technology can correct 13% of RPGR mutations in stem cells. Researcher Bassuk pointed out that the results of the study are very encouraging, because gene mutations are generally located in the highly repetitive sequence of the RPGR gene. In fact, it is determined that the specific DNA sequence in the gene is very specific. It is challenging, and researchers do not know whether CRISPR/Cas9 technology can correct point mutations in genes correctly.

　　CRISPR technology could not be used in human research before, because many scientists are worried that the technology will cause potential genetic mutations in the body, and the ethics society also believes that this technology will change human sperm and egg cells, and the researchers said that there are still A lot of work needs to be done. They hope that patients with special retinal degeneration diseases can be monitored and researched in real time to help develop new disease treatments.