The Grapevine

Scientists Use Gene Editing To Help HIV Patient Delete Disease In China

For the first time, scientists used the gene editing tool CRISPR-Cas9 in an attempt to treat an HIV patient in China. The patient showed improvements after the procedure and did not experience side effects. 

The scientists at Peking University in Beijing utilized CRISPR to delete the gene called CCR5 from stem cells in a donated bone marrow. CCR5 is known for contributing to HIV infection. 

A 27-year-old patient diagnosed with AIDS and acute lymphoblastic leukemia received the transplant. Doctors said the new and modified bone marrow should help treat his cancer and eliminate HIV. 

"After being edited, the cells -- and the blood cells they produce -- have the ability to resist HIV infection," lead scientist Deng Hongkui told CNN.

The patient went under the knife for the bone marrow transplant in 2017. In early 2019, scientists said the man's acute lymphoblastic leukemia “was in complete remission.”

The stem cells with the edited CCR5 gene also stayed in his system 19 months after the procedure. The team published the results in The New England Journal of Medicine.

However, the new cells did not completely eliminate the HIV virus. Scientists said the patient lacked enough amounts of stem cells to treat the disease. 

Cells in the transplanted bone marrow carried only 5 percent to 8 percent of the edited CCR5. But it might not be a major problem for future experiments since enhancing the gene editing technique may improve outcomes, the scientists said. 

"In the future, further improving the efficiency of gene-editing and optimizing the transplantation procedure should accelerate the transition to clinical applications," Deng said. 

The initial study mainly aimed to test the safety and feasibility of using genetically-edited stem cells for AIDS treatment. Deng said one key finding is that the procedure did not cause any negative effect. 

He added CRISPR has the potential to end blood-related diseases such as AIDS, sickle anemia, hemophilia and beta thalassemia. 

Deng’s team is not the first group to explore the use of CRISPR. China has been increasing its investment in the gene editing tool, which led to a number of first time experiments.

In 2016, the government announced biotechnology as part of its new Five-Year Plan. China is the first country to allow the use of CRISPR in humans and for the modification of nonviable human embryos.

Gene Editing CRISPR/Cas9 continues to provide scientists new ways to understand and fight previously untreatable diseases. Pixabay

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