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PHILADELPHIA – A new approach pioneered at the University of Pennsylvania’s Abramson Cancer Center may provide a new path towards treating Acute Myeloid Leukemia (AML) with CAR T cells. To treat AML, investigators have to target a specific protein – CD33 – that’s also expressed on healthy cells, meaning the therapy cannot attack the cancer without causing other serious damage to the residual normal bone marrow. The new method uses the gene editing tool CRISPR/Cas9 to remove CD33 from healthy blood-forming stem cells, leaving the cancerous cells as the only targets left for the CD33 hunter cells to attack. Penn researchers and their collaborators at the National Institutes of Health published their proof-of-concept findings in Cell today.

AML is the second most common type of leukemia, and the American Cancer Society estimates there will be almost 20,000 new cases in the United States this year. Many of these patients will undergo a bone marrow transplant. To treat a related leukemia called acute lymphoid leukemia (ALL), investigators at Penn previously developed CAR T cell therapy, which involves collecting patients’ own immune T cells, reprogramming them to kill cancer, and then infusing them back into patients’ bodies. Currently, both CAR T

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