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。
But there weren't enough of them to eradicate the HIV virus in the patient's body. After transplantation, only approximately 5% to 8% of the patient's 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.
【中国科学家基因编辑治疗艾滋病 向"治愈"又迈出一步】相关文章:
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