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Discovery And Research of AIDS Gene Therapy

Proposal of AIDS Gene Therapy

On October 28, according to media reports, the National Institutes of Health (NIH) announced yesterday that the agency will invest 100 million US dollars in the next four years to use gene therapy to cure AIDS and sickle cell disease (anemia). To this end, the National Institutes of Health will cooperate with the Bill and Melinda Gates Foundation, which will also invest $100 million in this project. The main goal of this cooperation is to enable people all over the world to afford treatment and have access to treatment, especially in developing countries where the burden of disease is the heaviest. The agency plans to prepare for clinical trials in the United States and sub-Saharan Africa within the next 7 to 10 years.

The Birth of AIDS Gene Therapy

At present, there are about 38 million people living with HIV in the world. Most of them live in developing countries. About two-thirds live in sub-Saharan Africa. The same is true for sickle cell disease. For decades, the National Institutes of Health has been trying to find a cure for AIDS. Although the currently used antiretroviral therapy can effectively suppress the virus in the body, it cannot really cure AIDS, and it must be taken daily to ensure the effect. In addition, as many as millions of people worldwide do not have the conditions to receive antiretroviral therapy.
Although scientists are developing gene therapies against HIV, these therapies are often costly and cannot be implemented on a large scale. For example, some therapies require cells to be removed from the patient's body and then reinjected into the patient's body, which is costly, time-consuming and long. For this reason, this new project will focus on the development of "in vivo" therapies, such as removing the CCR5 receptor gene from cells (the HIV virus uses CCR5 receptors to invade cells). Another idea is to try to remove HIV proviral DNA from cells. The HIV virus copies these DNA into the human cell genome, and even after receiving treatment, it can lie in it for several years.
The agency has set a similar goal for sickle cell disease: to repair the genetic mutation that causes the disease through in vivo therapy. This requires scientists to develop a gene-based drug delivery system that specifically targets variant genes for treatment. But to ensure that these new therapies are safe and effective, scientists still need to do a lot of work. Just a few months ago, the Trump administration just announced a plan to end the spread of HIV in the United States within 10 years.
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