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Reprogramming human T cell function and specificity with non-viral genome targeting

Summary of work

Parker Institute scientists at UCSF and UCLA developed a potentially more cost-effective and rapid way of performing gene editing, with significant applications for cell therapy and CAR-T immunotherapy treatments. The new method involves CRISPR and avoids the use of expensive viruses that can often create a bottleneck in cell therapy production. In this paper, PICI researcher Alexander Marson, MD, PhD, first used the technology to correct single disease-causing mutations in human cells. In a second application done in collaboration with Antoni Ribas, MD, PhD, Parker Institute director at UCLA, the researchers demonstrated it was possible to use the gene editing to insert large amounts of DNA into T-cells, altering them to more effectively target and kill tumor cells.

Why this is impactful to patients

“This is a huge advance for the cell therapy and CAR-T field, opening the door for us to create more robust, personalized cancer immunotherapy treatments in less time,” said Fred Ramsdell, PhD, vice president of research at the Parker Institute. “What takes months or even a year may now take a couple weeks using this new technology. If you are a cancer patient, weeks versus months could make a huge difference.”