Chinese scientists successfully repaired Marfan syndrome gene mutation for the first time in the world
September 10, 2018 Source: Ministry of Science and Technology
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 August 14th, the international academic journal Molecular Therapy published the research team of Professor Liu Jianqiao from the Third Affiliated Hospital of Guangzhou Medical University and the team of Professor Huang Xingxu of Shanghai University of Science and Technology to repair genetic disease Ma Fan syndrome at the embryonic level. the latest results prove the efficiency and safety of base editing techniques on human embryos for disease-causing mutation repair.
Marfan syndrome is an autosomal dominant genetic disease (genetically only on the key gene of "FBN1", the position of A and G has been changed), the patient's limbs, fingers, and toes are slender and uneven, so Known as the "spider man." At present, there is no specific drug for this disease, and surgery can only alleviate the symptoms and is expensive. Most of the patients died in their 50s.
The research team uses a Harvard University David Liu developed in the laboratory in 2016 base editor, directly to the "FBN1" mutant gene in position A and G are swapped "homing" corrected "spelling errors" in the final Among the 18 embryos, 16 completely normal genotypes were obtained, which successfully blocked this genetic disease. The world's first use of base editing technology successfully repaired the pathogenic genes of Marfan syndrome at the embryo level.
The research results have been paid attention to by many foreign media such as Science magazine. The famous American technology magazine "Connecting" and the developers of the technology have highly praised this achievement, but Professor Liu Jianqiao said: "This is The treatment of single-base mutation genetic diseases provides a possible treatment option, but due to ethical, reliability, safety and other issues and related laws and regulations, this technology is still a long way from clinical application."
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