CRISPR/Cas9技术,艾滋病 z-bo 2020-10-14 371

　　HIV (HIV) is an effective and cunning retrovirus. When HIV introduces its DNA into the genome of the host cell, it can incubate for longer periods of time and can remain dormant for several years. Doctors can prepare a matching mixture of antiretroviral drugs to control the virus, but if treatment is discontinued, the virus will reappear.

　　In order to make the potential HIV completely harmless, researchers at the University of Massachusetts Medical School can use the powerful gene editing tool Cas9/CRISPR to transform the potential viral DNA. A new technique has been developed to remove infected cells. Co-author of the study, Scott Wolf, associate professor of molecular, cell and cancer biology at the University of Massachusetts School of Medicine, said: The end of the genome. If this can be done, it will be a step towards functional treatment of AIDS. CRISPR is part of the normal bacterial immune system. In its natural state, it can protect bacteria. Invaded by a virus. Since its discovery, researchers have been looking for ways to design the system to quickly and selectively edit specific gene sequences for research.

　　CRISPR system is widely used, but its application is still limited to laboratories. Recent studies have shown that Cas9/CRISPR can edit HIV in cultured infected cells, but the accuracy of this technology usually cuts into random regions of the genome, leading to harmful off-target effects. It is not enough for clinical application.

　　Cas9 / In order to improve the fidelity and accuracy of the CRISPR gene editing system, Wolfe proposed to fuse it with another domain to improve its specificity. This will allow the CRISPR system to edit only HIV DNA, making it impossible to accidentally delete the human genome.

　　Another obstacle to using the existing Cas9/CRISPR technology to fight AIDS is that researchers believe that the virus may be hidden in several places, but I still don’t know how to find the virus lurking in infected cells. Dr. Jeremy Ruban, professor of molecular medicine at the University of Massachusetts School of Medicine, said: "HIV-infected cells are the permanent vectors of the viral genome. Whenever patients stop taking antiretroviral drugs, they become time bombs. They are. It can relapse. You really need to understand where you live and what you need to survive."

　　Uban and Wolf use innovative techniques to describe and simulate HIV integrated into the genome of reservoir cells. Do it. Viral DNA, also known as provirus. The genomic blueprints that characterize these potentially infected cells allow researchers to identify vulnerable gene sequences.

　　Luban said: “Many scientists are looking for tools to activate the virus so that it can be recognized by the immune system and drugs. We hope to isolate and remove the original virus directly from dormant cells. We chose another method.”

　　Using the genome of potentially infected cells as a model, Wolfe hopes to use his precise gene editing tools to eliminate viruses lurking in the cells. I will. Part of the project is whether the accuracy of the system has been improved to the extent that it is sufficient to selectively remove the HIV genome from humanized mouse models and infected patient cells without indirectly destroying the human genome. survey

　　Luban said: "The basic premise of the project is to use genetics to design a system that can remove the HIV genome from infected cells. We hope to develop tools to deliver these drugs to the human immune system. In fact, in cells, The virus is cleaned from its hidden parts,"

　　Wolff explained. complex structure. We believe that this will allow us to use CRISPR to better target viruses for gene editing. "