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No Side Effects in Small Trial

Advanced cancer patients treated with immune cells that underwent CRISPR-mediated gene editing exhibited no serious side effects, New Scientist reports.

It notes that concerns about the treatment particularly revolved around whether unintended CRISPR cuts occur and turn those cells cancerous as well.

The University of Pennsylvania's Carl June and his colleagues conducted a first-in-human phase I clinical trial examining the safety and feasibility of using CRISPR-Cas9 to engineer T cells from three advanced cancer patients. As they report this week in Science, the researchers found that the modified T cells — they deleted three genes and added one — persisted in the patients for at least nine months and that the treatment appeared safe.

"Before we did this, no one had ever infused CRISPR-edited cells into patients, and we're encouraged by the fact that we could do it safely," first author Edward Stadtmauer, an oncologist at UPenn, tells Wired. "Now we can move on to a whole new frontier of further engineering these cells and expanding the number of patients treated."

New Scientist notes, though, that the researchers are eyeing a new CRISPR technology for their follow-up studies. June tells it that they are exploring CRISPR base editing as it can inactivate genes without cutting, which lessens the chance of the treatment leading to cancer.