Genetic Engineering May Reduce CAR T Treatment Toxicity in AML

Using gene editing to remove CD33, a protein targeted to treat acute myeloid leukemia with chimeric antigen receptor (CAR) T cells, from healthy stem cells could reduce toxicity experienced by CAR T-cell therapy, according to a study

published in Cell

.

A group of researchers at the University of Pennsylvania (Penn) and collaborators at the National Institutes of Health hypothesized that deleting CD33 from healthy cells could create an antigen that only targets the cancerous cells.

Previous attempts to target CD33 with CAR T therapies have damaged healthy cells. When CAR T cells are used short term, the damage is prevented, but that goes against the purpose of the treatment, which is to ensure CAR T cells remain in circulation within the body for years, thereby preventing relapse.

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