中国科学家完成了世界首例基于基因编辑干细胞治疗艾滋病和白血病的案例。消息一出,随即引发全球关注,也让很多患者燃起了新的希望。
The 27-year-old patient's prospects were bleak. In May 2016, he found out he had AIDS. Two weeks later, he was told he had acute lymphoblastic leukemia.
这位27岁的病人前景黯淡。2016年5月,他发现自己患有艾滋病。两周后,他被告知患有急性淋巴细胞白血病。
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.
但根据《新英格兰医学杂志》最新发表的一篇论文,医生们给这位中国公民带来了一线希望:通过骨髓移植来治疗他的癌症,并进行另外的实验性治疗,试图清除他体内的艾滋病病毒。
bone marrow transplant:骨髓移植
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.
参与研究的北京大学科学家说,治疗手段包括使用基因编辑工具CRISPR-Cas9从捐赠者的骨髓干细胞中删除一种名为CCR5的基因,然后将干细胞移植到患者体内。
"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.
“经过编辑后,这些细胞——以及它们产生的血液细胞——有能力抵抗艾滋病毒感染,”首席科学家邓宏魁在上周五(9月13日)告诉美国有线新闻网。
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 person's 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.
携带有发生突变的CCR5基因拷贝的人对艾滋病毒具有很高的免疫力,因为艾滋病毒利用这种基因产生的蛋白质进入感染者的细胞。“柏林患者”和“伦敦患者”在接受了来自天生携带有这种基因突变的捐赠者的骨髓移植后,成为世界上首批被治愈的艾滋病毒感染者。
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.
在患者同意后,实验于2017年夏天进行。这是基因编辑工具CRISPR-Cas9首次用于艾滋病毒患者。2019年初,也就是接受治疗整整19个月后,“急性淋巴细胞白血病完全缓解,携带有经过编辑的CCR5基因的供体细胞持续存在,”科学家在论文中说。
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