CRISPR/Cas9n编辑,帕金森病 z-bo 2020-10-26 363

　　In the UK, the incidence of Parkinson's disease is about 1 in 350. In this new study, researchers from the University of Edinburgh and UCB Pharmaceuticals (UCBPharma Ltd.) have taken an important step in improving new treatments for Parkinson's disease. This progress may contribute to the development of a promising treatment method called cell replacement therapy (cell replacement therapy).

　　Experts hope this therapy will transplant healthy cells into the brain area destroyed by Parkinson's disease, thereby reducing symptoms such as tremor and balance problems. In this new study, these researchers constructed human embryonic stem cells (hESC) that are resistant to Parkinson's disease. Specifically, we used an advanced technology called CRISPR/Cas9n to cut hESC DNA fragments. By doing so, they eliminated the SNCA gene associated with the formation of toxic substances (called Rui bodies), which is a typical feature of brain cells in Parkinson's patients. In laboratory tests, these stem cells can be transformed into dopamine-producing neurons in a petri dish. Then it is treated with chemicals to form a Rui body. These researchers found that compared with non-gene-edited neurons, gene-edited neurons did not form toxic blocks. These researchers say that this advancement may be most beneficial to young patients with Parkinson's disease and patients with aggressive Parkinson's disease, but it still needs to be tested in human trials.

　　The corresponding author of this article and Dr. Tiro Kunas of the Centre for Regenerative Medicine, University of Edinburgh Medical Research Council, said: "Parkinson's disease spreads between neurons and invades healthy cells. Deadline. Our exciting findings have the potential to improve significantly. These new treatments."

　　Parkinson’s Disease Cure Parkinson’s Trust Research Deputy Director Dr. Simonstott said: Experimental regenerative medicine for patients. This new research has brought new progress to the development of the therapy. We are pleased that the Parkinson's Disease Treatment Foundation has provided funding for this exciting and innovative research. "