The world's first human genome editing test implementation
November 20, 2017 Source: Technology Daily
Window._bd_share_config={ "common":{ "bdSnsKey":{ },"bdText":"","bdMini":"2","bdMiniList":false,"bdPic":"","bdStyle":" 0","bdSize":"16"},"share":{ }};with(document)0[(getElementsByTagName('head')[0]||body).appendChild(createElement('script')) .src='http://bdimg.share.baidu.com/static/api/js/share.js?v=89860593.js?cdnversion='+~(-new Date()/36e5)];On the 17th, the online edition of Science magazine reported a milestone in the history of human medical treatment: scientists first attempted to directly edit genes in the human body. They injected a genetic editing tool into the blood of a 44-year-old patient to cure severe genetic diseases by permanently altering the genes.
The clinical trial was completed at the University of California, San Francisco, and the subject was Brian Mard, a 44-year-old Hunter's syndrome patient. Hunter's syndrome is a rare, life-threatening genetic disease caused by genetic mutations. The patient's cellular metabolic waste cannot be decomposed and accumulates in tissues and organs, eventually resulting in dysfunction.
The genetic editing tool that Mader accepts is zinc finger nuclease, which is not a popular concern. Compared with the latter, zinc finger nuclease has a higher "qualification". It is considered to be the first generation of gene editing tools. The sequence for positioning is longer, the operation is relatively complicated, and the precision of gene editing is higher.
In this treatment, the non-pathogenic adeno-associated virus will carry two zinc finger nucleases and a normal gene, directly to the patient's liver cells. Upon arrival, the zinc finger nuclease will accurately find the "working position" and cut the DNA duplex into a normal gene like a pair of scissors. Through the DNA self-healing mechanism, the original DNA fragment will accept new normal genes.
In the previous "gene therapy", scientists edited human cells in a culture dish and injected them back into the human body. They did not directly edit the patient's DNA. Previously, there have been problems with limited treatment and partial effects. The new treatment results will get the exact answer within three months, and once successful, it will push the gene therapy into a comprehensive stage.
The trial currently raises some concerns: although zinc finger nuclease safety has been tested and animal models have received good feedback, there are still unpredictable side effects. However, officials from the National Institutes of Health who participated in the approval of the project said that so far there is no evidence that the therapy is dangerous and therefore should not be afraid. (Reporter Zhang Mengran)
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