Gene-edited stem cells may help cure AIDS
2019-09-26 11:20

Gene-edited stem cells that are the precursors of blood cells may help cure AIDS patients, a new discovery by Chinese scientists shows, providing new insights into treatment of the serious infectious disease.


In the study, researchers used the stem cells and other progenitor cells that had been gene edited to reduce a protein called CCR5, which serves as a doorway for HIV infection of human cells. The gene-edited cells were transplanted into a patient infected with HIV and with acute lymphoblastic leukemia, a common type of leukemia.

在这项研究中,研究人员使用经过基因编辑的干细胞和其他祖细胞来减少被称为CCR5的蛋白质——该蛋白质是HIV 感染机体细胞的重要辅助受体。经过基因编辑的干细胞被移植到感染了 HIV 和急性淋巴细胞白血病(一种常见的白血病)的患者体内。

The patient, a 27-year-old Chinese, improved greatly during a 19-month follow-up period and showed almost no symptoms of acute leukemia. In addition, the transplanted gene-edited cells showed resistance to HIV infection during a brief period when the patient stopped taking antiviral drugs, according to the study, published in the New England Journal of Medicine on Sept 11.

该患者是一名27岁的中国人,经过19个月的随访,患者病情得到了很大的改善,几乎没有出现急性白血病的症状。此外,这项发表在9月11日《新英格兰医学杂志》上的研究表明,在患者短暂停用抗病毒药物期间,移植的基因编辑干细胞表现出了抵御 HIV 感染的能力。

Deng Hongkui, a professor in life sciences at Peking University, and a chief researcher involved in the study, said researchers overseas have been trying to use gene-edited stem cells to treat AIDS patients, but the study is the first to have gained initial success in clinical trials of the new methods. The study started in May 2017 and is continuing, he said.


"The study indicates great potential for gene-editing technologies in the treatment of serious diseases, including AIDS, hemophilia and thalassemia," he said. Thalassemia is an inherited disease in which people have an overload of iron in their bodies.


Previously, scientists from abroad succeeded in treating AIDS patients with transplanted bone marrow cells with a natural genetic mutation in the CCR5 protein that made it immune to HIV infection in the few reported cases. A major reason that such treatment is uncommon is that the genetic mutation is rare among humans, which makes finding the right donor extremely difficult.

此前,国外的科学家曾成功地用移植的骨髓细胞治疗了艾滋病患者,该骨髓细胞的CCR5蛋白发生自然遗传突变,从而使其成为少数报道中对 HIV 免疫的例子。但由于人类很少发生基因突变,寻找合适的供体非常困难,使得这种治疗方法极为罕见.

A new report involving research led by the University of Cambridge was published in March in Nature. A patient with HIV showed immunity to the virus after receiving bone marrow stem cells from such a donor.


Although researchers achieved successful transplantation and long-term grafts of stem and progenitor cells using the CRISPR gene-editing technique, the efficiency of gene editing using the CCR5 protein is not high, indicating the need for further research into this approach, Deng said.


"The research explored the feasibility and safety of the method," he said. "We need to further improve the efficiency of gene editing and optimize transplantation procedures in our future study, and it is expected that application of the gene-editing technology to clinical use to treat diseases will be accelerated," he said.


Although no adverse results related to the gene editing have been seen in the research, long-term, intensified research is still needed to evaluate the safety of the technology, he said.


The number of people living with HIV worldwide is estimated at 37 million, according to the Joint United Nations Programme on HIV/AIDS.


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