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With genetic tweaks and snips, researchers created cancer-busting immune cells that, so far, seem to have wiped out a life-threatening form of leukemia in a one-year-old girl.
The T cells she received, engineered by Waseem Qasim of the University College London and his team, were modified to have an extra gene and two broken ones. The extra gene encodes a receptor called CAR19, which allows the T cell to recognize and kill cancer cells. The researchers broke two genes using a molecular method, called TALEN (transcription activator-like effector nucleases), that snips and inactivates genes. One slicing took out a gene for a receptor so that the engineered T cells wouldn’t attack healthy cells in the patient. The other gene slashing made the T cells immune to a powerful leukemia drug.