• Using their novel approach, the scientists 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. Scientists believe the development could eventually result in a new approach to fighting viral infections and cancerous tumors.
  • Until recently, editing human T cells with CRISPR/Cas9 has been inefficient, with only a relatively small percentage of cells being successfully modified. And 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.
  • "I think CRISPR-edited T cells will eventually go into patients, and it would be wrong not to think about the steps we need to take to get there safely and effectively." said Alexander Marson, PhD, a UCSF Sandler Fellow, and senior and co-corresponding author of the new study.

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