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中国科学家基因编辑治疗艾滋病 向治愈又迈出一步

行业资讯 / 2021-12-01 00:07

本文摘要:The 27-year-old patients prospects were bleak. In May 2016, he found out he had AIDS. Two weeks later, he was told he had acute lymphoblastic leukemia.这名28岁的患者市场前景暗淡。二零一六年五月,他察觉自己得了HIV。两个星期后,他被告知得了亚急性网织红细胞败血症。


The 27-year-old patients prospects were bleak. In May 2016, he found out he had AIDS. Two weeks later, he was told he had acute lymphoblastic leukemia.这名28岁的患者市场前景暗淡。二零一六年五月,他察觉自己得了HIV。两个星期后,他被告知得了亚急性网织红细胞败血症。

But doctors offered the Chinese citizen a ray of hope: a bone marrow transplant to treat his cancer and an extra experimental treatment to try to rid his system of HIV, according to a new paper published in The New England Journal of Medicine.但依据《新英格兰医学杂志》最近公布发布的一篇毕业论文,医生和护士给这名中国人带来了一线希望:根据骨髓移植来化疗他的癌病,并进行此外的试验性化疗,妄图清除他身体的艾滋病毒。This involved using the gene editing tool CRISPR-Cas9 to delete a gene known as CCR5 from bone marrow stem cells taken from a donor, before transplanting them into the patient, Peking University scientists said in the study.参与科学研究的北大生物学家说道,化疗方式还包含用以基因编辑专用工具CRISPR-Cas9从捐赠人的骨髓干细胞中清除一种起名叫CCR5的遗传基因,随后将干细胞美容重制到病人身体。

After being edited, the cells -- and the blood cells they produce -- have the ability to resist HIV infection, lead scientist Deng Hongkui told CNN Friday.“历经编写后,这种体细胞——及其他们造成的血夜体细胞——有工作能力抵御艾滋病毒病毒性感染,”首席科学家邓宏魁在上周五(9月13日)对他说英国有线电视新闻。People who carry defective copies of CCR5 are highly immune to HIV, because the virus uses a protein made by this gene to gain entry into an infected persons cells. Two men, known as the Berlin patient and the London patient, became the first people in the world to be cured of HIV after receiving bone marrow transplants from donors who had the mutation naturally.装车有再次出现基因变异的CCR5遗传基因复制的人对艾滋病毒具有很高的免疫能力,由于艾滋病毒运用这类遗传基因造成的蛋白转到病毒感染者的体细胞。


“纽约病人”和“纽约病人”在拒不接受了来源于与生俱来装车有这类基因变异的捐赠人的骨髓移植后,沦落全世界第一批被治疗的艾滋病毒病毒感染者。The patient agreed and the experiment was carried out in the summer of 2017. It was the first time CRISPR-Cas9 was used on a HIV patient. In early 2019, a full 19 months after the treatment was administered, the acute lymphoblastic leukemia was in complete remission and donor cells carrying the ablated CCR5 persisted, the scientists said in the paper.在病人完全同意后,试验于17年夏季进行。

它是基因编辑专用工具CRISPR-Cas9初次作为艾滋病毒病人。今年初,也就是拒不接受化疗整整的19月后,“亚急性网织红细胞败血症放任不管,装车有历经编写的CCR5遗传基因的供细胞不断不会有,”生物学家在毕业论文中说道。But there werent enough of them to eradicate the HIV virus in the patients body. After transplantation, only approximately 5% to 8% of the patients bone marrow cells carried the CCR5 edit, according to the researchers. In the future, further improving the efficiency of gene-editing and optimizing the transplantation procedure should accelerate the transition to clinical applications, said Deng.但这还足够击溃患者身体的艾滋病毒。


科学研究工作人员说道,重制后,仅有约5%到8%的病人骨髓细胞装车这类历经编写的遗传基因。邓宏魁说道,“在未来,进一步提高基因编辑的高效率和提升重制程序流程等对策理应不容易加速向临床医学运用于的更改。”But he doesnt see this as a setback.The main purpose of the study was to evaluate the safety and feasibility of genetically-edited stem cell transplantation for AIDS treatment, said Deng.但他不强调这一化疗是一次结束。

“此项科学研究的关键目地是评定基因编辑干细胞美容重制化疗HIV的安全系数和可行性分析,”邓宏魁说道。According to Deng, this was a success: the scientists didnt detect any gene editing-related adverse events, even if more long-term in-depth studies are needed for off-target effects and other safety assessments, Deng said.他强调这一实验很成功:专家没找到一切与基因编辑涉及到的不善恶性事件,邓宏魁说道,即便“务必更为长时间掌握的科学研究来进行脱靶效应和别的安全系数评定。”The CCR5 gene mutation has been associated with a 21% increased risk of dying early, according to a paper published in Nature in June, though its unclear why.《大自然》杂志期刊2020年6月公布发布的一篇毕业论文称作,CCR5基因变异与早逝风险性降低21%相关,但是缘故行远必自不准确。

The team that conducted the study had previously transplanted edited CCR5 human cells into mice, making them resistant to HIV. American scientists have carried out similar experiments on humans, with some success, using an older gene editing tool called zinc finger nuclease.进行此项科学研究的精英团队先前曾将CCR5编写过的人们体细胞重制到小白鼠身体,使其对艾滋病毒具有抵抗能力。美国科学家早就在人们的身上进行了类似的试验,并得到 了一些成功,她们用以了一种更为历史悠久的称之为锌指核酸酶技术性的基因编辑专用工具。China has invested heavily in gene-editing technology, making biotech one the priorities of its Five-Year Plan announced in 2016. The central government has bankrolled research into a number of world firsts, including the first use of the gene-editing tool CRISPR-Cas9 in humans in 2016 and the first reported use of gene editing technology to modify nonviable human embryos in 2015.我国在基因编辑技术性上推广重金,将生物科技纳入二零一六年宣布的五年规划(十三五规划)的关键之一。我国政府支助了多种可以说全球“第一”的科学研究,还包含二零一六年初次在人们的身上用以基因编辑专用工具CRISPR-Cas9,及其二零一五年初次用以基因编辑技术性修改没法生存的人们试管胚胎。

Deng Hongkui remains a strong believer in CRISPR-Cas9. He thinks it could bring a new dawn to blood-related diseases such as AIDS, sickle anemia, hemophilia and beta thalassemia and that, thanks to this new technology, the goal of a functional cure for AIDS is getting closer and closer.邓宏魁仍然是基因编辑专用工具CRISPR-Cas9的忠实教徒。他强调这将为血夜涉及到病症带来“新的曙光”,例如HIV、镰状体细胞缺铁性贫血、血友病甲和乙型肝炎地中海贫血症。并且,因为此项新技术应用,“大家间距多功能性治疗HIV的总体目标更为类似”。