Health
Bold Efforts to Cure HIV—With Crispr
Dornbusch believes this strategy protects patients from serious side effects and “off-target” editing (unintended cuts elsewhere in the genome that can cause problems such as cancer).
The region targeted by the company’s Crispr therapy is also part of the genome that tends to remain unchanged as HIV evolves. This is important because viruses mutate rapidly and researchers don’t want moving targets.
It’s not the first time scientists have tried gene editing in hopes of curing people infected with HIV, but other studies have shown CCR5In the 1990s, scientists discovered that people with this naturally occurring mutation did not get HIV when exposed to it. This mutation, known as delta 32, interferes with the virus’ ability to enter immune cells. In 2009, California-based Sangamo Therapeutics used an old editing technique called zinc finger nuclease to add protective mutations to a patient’s T cells, a vital part of the immune system.those trials limited success.
In 2017, Chinese scientists combined Crispr with a bone marrow transplant. try to cure HIV and leukemia patientsIn a typical transplant, a donor’s stem cells are transplanted into the recipient to replace the cancerous blood cells. These cells continue to form new healthy blood cells.To also address HIV in patients, researchers edited donor stem cells with Crispr to disable them CCR5After transplantation, however, only a small fraction of the patient’s bone marrow cells finished the desired editing.
Then, in 2018, Chinese scientist He Jiankui used Crispr to CCR5 mutation to Genomics of twin girls to make them resistant to HIV.full ethical violationthe experiment is Widely condemned by scientistsHis research was stopped by the Chinese government and he served a three-year prison sentence. The twins were born healthy, but only some cells were successfully edited.
2 as of 2022 HIV is now cured after receiving a bone marrow transplant from a donor CCR5Known as the Berlin Patient, patient in london, both had cancer and received transplants to treat the disease. However, these transplants are not viable options for most people. Few donors are at high risk and carry delta32 mutations.However, a third party Declared cured of HIV She died earlier this year after undergoing a new type of transplant containing cord blood.
The Excision trial will eventually enroll nine participants and test three doses to determine which is most effective. The investigator will measure each person’s viral load and her CD4 count before receiving treatment and after stopping antiretroviral drugs. The ultimate goal is to reduce the viral load to undetectable levels. That is, less than 200 copies of HIV per milliliter of blood. At this level, HIV cannot be transmitted through sex.
Excision’s challenge is to get Crispr to reach enough cells to reduce HIV to undetectable levels. The company uses genetically engineered viruses to deliver gene-editing components to a patient’s HIV-infected CD4 cells. However, so far there is little human data on how well Crispr works when delivered directly to the body. , we may be able to get the virus to levels so low that people don’t need to take antiretroviral therapy,” said Rowena Johnston, Associate Director of Research at amfAR, the AIDS Foundation.
And while these drugs are highly effective, many people would rather be completely free of the virus, says Johnston. “People with HIV still live with a lot of stigma and internalized shame,” she says. “I think that therapy, no matter how easy it becomes, is much better than lifelong therapy.”
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