艾滋病有望被治癒!基因編輯成功清除小鼠體內HIV病毒大綱

Researchers who eliminated HIV from mice say their results are an important advance in efforts to develop a cure for HIV infection in people.

研究人員近日清除了小鼠體內的艾滋病毒，並表示這項研究結果是在研究治癒人類艾滋病毒感染方面取得的重要進展。

This is the first time that the AIDS-causing virus has been eradicated from the genomes of living animals, according to the authors of the study.

研究報告的作者稱，這是人類首次從活體動物的基因組中完全消除艾滋病毒。

"Our study shows that treatment to suppress HIV replication and gene editing therapy, when given sequentially, can eliminate HIV from cells and organs of infected animals," said co-senior author Kamel Khalili, professor and chair of neuroscience at Temple University in Philadelphia.

“我們的研究表明，循序進行抑制艾滋病毒複製的治療和基因編輯治療可以消除被感染動物體內細胞和器官中的艾滋病毒”，費城天普大學神經科學教授兼主任、論文資深合著者卡邁勒·卡利利說。

The work was a collaboration with researchers at the University of Nebraska Medical Center.

這項工作是與內布拉斯加大學醫學中心的研究人員合作完成的。

Current HIV treatment uses antiretroviral therapy (ART), which suppresses HIV replication but does not eliminate the virus from the body. ART is not a cure for HIV and requires lifelong use.

目前的艾滋病毒治療使用抗逆轉錄病毒療法，可以抑制艾滋病毒的複製，但無法將病毒從體內消除。抗逆轉錄病毒療法不能治癒艾滋病毒，需要終生使用。

In this study, the researchers used a gene editing system called CRISPR-Cas9 to remove large fragments of HIV DNA from infected cells, along with a new drug regimen called long-acting slow-effective release (LASER) ART.

在這項研究中，研究人員使用了一種名爲CRISPR-Cas9的基因編輯系統，從受感染的細胞中去除較大的艾滋病毒脫氧核糖核酸片段，同時使用了一種名爲長效緩釋（激光）的新藥療法。

In this therapy, antiretroviral drugs are placed in nanocrystals that travel to tissues where HIV is likely to be lying dormant. The nanocrystals are stored within cells for weeks and slowly release the drugs.

在治療過程中，抗逆轉錄病毒藥物被放置在納米晶體中，並被運送到可能潛伏着艾滋病毒的組織。納米晶體被儲存在細胞內數週，緩慢釋放藥物。

Mice with HIV were first treated with LASER ART and then with gene editing. This approach eliminated HIV DNA from about one-third of the mice. However, research on animals may not produce the same results in humans.

研究人員首先用激光抗逆轉錄病毒療法治療感染艾滋病毒的小鼠，之後使用基因編輯技術。這種治療方法消除了約三分之一小鼠體內的艾滋病毒脫氧核糖核酸。然而，對動物的研究可能不會在人類身上達到相同的效果。

"The big message of this work is that it takes both CRISPR-Cas9 and virus suppression through a method such as LASER ART, administered together, to produce a cure for HIV infection," Khalili said in a university news release.

卡利利在一份大學新聞稿中說:“這一研究的重要信息是，它需要使用CRISPR-Cas9基因編輯，並通過激光抗逆轉錄病毒療法等方法抑制病毒，兩種方法一起使用，才能治癒艾滋病毒感染。”

"We now have a clear path to move ahead to trials in nonhuman primates and possibly clinical trials in human patients within the year," added Khalili, who also directs Temple's neurovirology center and its neuroAIDS center.

“現在我們有一條清晰的道路，我們可以在年內對靈長類動物進行試驗，也可能對艾滋病感染者進行臨牀試驗”，同時主管天普大學神經病毒學中心和神經艾滋病中心的卡利利補充說。

The results were published online July 2 in the journal Nature Communications.

該研究結果於7月2日發表在《自然通訊》雜誌網絡版上。