'Because of this study, we now know that these edited cells can survive in a patient, and they will stay there,' an expert said.
gene-editing technique to try to cure a man of HIV, in what one expert called an"important step" forwards in treating disease.
Hailed as a revolutionary leap forward for conducting research and finding potential treatments for disease, CRISPR makes it quicker and easier to edit DNA. The technique called CRISPR-Cas9 involves using the Cas-9 protein to snip at a specific part of DNA.to edit a donor's stem cells and transplant them into a 27-year-old patient with HIV and leukemia. They hoped the cells would survive, replicate, and cure the man of HIV.
The approach involved knocking out the CCR5 gene in the donor's stem cells. CCR5 encodes a protein that HIV uses to get inside human blood cells—and past research has shown those with a mutation on the gene are protected from HIV.gene-edited cells were still working 19 months after they were transplanted. However, they made up five to eight percent of the recipient's stem cells. Just over half of the edited cells had died away.
The team were pleased to find the man didn't appear to suffer any adverse effects linked to the transplant. The findings were published in theThe work was inspired by the treatment of Timothy Ray Brown. Known as the Berlin Patient, Brown had leukemia and HIV and received a stem cell transplant to treat his cancer in 2007, and once more in 2008. His donor had a mutation on the CCR5 receptor, which makes an individual immune to HIV.
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