Investigators from the University of California San Francisco (UCSF) have developed a new strategy to precisely modify human T cells using CRISPR/Cas9—implications that could have sweeping impacts on AIDS, cancer, and various other autoimmune disorders.
“Genome editing in human T cells has been a notable challenge for the field,” explained senior author Alexander Marson, Ph.D., a Sandler Fellow at UCSF. “So we spent the past year and a half trying to optimize editing in functional T cells. There are a lot of potential therapeutic applications, and we want to make sure we’re driving this as hard as we can.”
The UCSF researchers were able to deactivate a protein on the surface of T cells called CXCR4, which is often exploited by the HIV virus to infect the T cell. Additionally, the scientists were able to nullify the PD-1 protein, which has made a big splash in the newly flourishing field of cancer immunotherapy, since blocking this protein with newly developed drugs has been shown to cause T cells to attack tumors.
The CRISPR technique has captivated the imaginations of scientists over the past several years since it makes it possible to efficiently and inexpensively edit genetic information in virtually any organism. T cells are a high valued target for medical applications of the technology, as these cells not only stand at the epicenter of many disease processes, but can be easily gathered from patients, edited with CRISPR/Cas9, then returned to the body to exert therapeutic effects.
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