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Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells

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Science Translational Medicine  25 Jan 2017:
Vol. 9, Issue 374, eaaj2013
DOI: 10.1126/scitranslmed.aaj2013

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CAR sharing

Chimeric antigen receptor (CAR) T cells can be very effective in treating acute lymphocytic leukemia. Unfortunately, these therapeutic cells have to be custom-made for each patient, and this is not always feasible, especially for patients who do not have sufficient healthy T cells. Qasim et al. demonstrate that there may be another option for these patients. By using gene editing to simultaneously introduce the CAR and disrupt TCR and CD52 in T cells, the authors generated functional CAR T cells that could evade host immunity for use in unmatched recipients. These “off-the-shelf” CAR T cells were then used to treat two infants with relapsed refractory acute lymphocytic leukemia and bridge them to allogeneic stem cell transplantation.