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“Technically speaking, this is the first use of CRISPR in human patients [in the United States], but CRISPR is an add-on to this therapy,” says John Doench, a Broad Institute geneticist and gene editing expert. CRISPR is just a new tool for conducting a slightly-less-new procedure. In the University of Pennsylvania trial, he says, CRISPR is being used to make engineered immune cells known as CAR-T cells which are thought to be better at attacking tumors that regular immune cells.
Other teams have made these engineered immune cells using other methods before, but CRISPR allows the University of Pennsylvania team to make a new alteration: knocking out a specific gene in the CAR-T cells related to suppressing their immune response. This gene, known as PD-1, can be targeted throughout the body with drugs called PD-1 inhibitors, but using CRISPR, the scientists can just modify the immune response of the special immune cells they’re creating. That decreases the side effects of the treatment: after all, PD-1 is in our bodies for a reason.
CAR-T - Cells - Technology - Doench - Therapy
“I think it’s important to recognize that CAR-T cells are a technology in themselves,” says Doench. That means if the therapy just doesn’t work for the two patients currently enrolled in the CRISPR trial, the reason may not be related to CRISPR at all; CAR-T is incredibly effective in some patients, but useless in others. Both CRISPR and CAR-T cells have a long way to go as therapies, and it’s possible that they just won’t work, or won’t work in combination, he...
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