Researchers at the University of California – San Francisco used the genome-editing tool known as CRISPR/Cas9 to precisely modify human T cells. They were able to disable a protein on the T-cell surface called CXCR4, which can be exploited by HIV when the virus infects T cells and causes AIDS.
The team also successfully shut down PD-1, a protein that has attracted intense interest in the burgeoning field of cancer immunotherapy, as scientists have shown that using drugs to block PD-1 coaxes T cells to attack tumours.
While scientists have had some success in switching off genes by inserting or deleting random sequences, they have not yet been able to use CRISPR/Cas9 to paste in (or 'knock in') specific new sequences to correct mutations in T cells.
A team led by first authors Kathrin Schumann, a postdoctoral fellow in the lab of Alexander Marson, a UCSF Sandler Fellow, and Steven Lin, a postdoctoral fellow in the lab of University of California, Berkeley's Jennifer Doudna, cracked these problems by streamlining the delivery of Cas9 and single-guide RNA to cells.


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