Using genetic editing technology to eliminate HIV in humans

Hu Wenhui, a Chinese scientist at Temple University in the United States, recently reported that they use gene editing technology to effectively eliminate human HIV in multiple organ tissues of humanized mice, and take a step toward human clinical trials. Big step.

Previously, Hu Wenhui and others have successfully used gene editing technology to effectively eliminate human cell lines cultured in vitro, T immune cells taken out from AIDS patients, and HIV in transgenic mice.

利用基因编辑技术可剔除人类体中艾滋病病毒

Professor Hu Wenhui, the head of the study (Source: Temple University)

Humanized BLT mice refer to immunodeficient mice transplanted with human bone marrow, liver and thymus tissue or cells. The mouse has a human functional immune system, which is consistent with humans by HIV infection and latent methods. It overcomes the drawbacks of conventional mice that cannot replicate certain human diseases and is widely used in AIDS animal experiments.

In a recent study published in the American Journal of Molecular Therapy, Hu Wenhui and fellow colleague Kamal Khalili and Pittsburgh University Yang Wenbin first used HIV to infect humanized BLT mice and then used adeno-associated virus. (AAV) As a vector, a CRISPR/Cas9 gene editing tool called "gene scissors" was transported to latent infected mice. Two to four weeks later, they detected a deletion of the HIV genome in multiple mouse organ tissues.

Associate Professor Hu Wenhui told reporters that the HIV gene is prone to mutation, and virus escape may occur with single-target gene editing. To this end, they proposed a new idea of ​​multi-target gene editing, designed four guide RNA (ribonucleic acid) for HIV transcribed regions and structural regions, which can guide Cas9 enzyme to a predetermined position to achieve multi-target resection, significantly increased HIV removal efficiency.

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