AAVS1 safe-harbor control CAR-T cells

AAVS1 safe-harbor control CAR-T cells are an experimental cell therapy used as a benchmark in genomic engineering studies to optimize CAR-T cell efficacy. Developed for research presented by Beatriz Coutinho De Oliveiro and colleagues, these cells are generated by electroporating healthy donor T-cells with Cas9 to disrupt the Adeno-Associated Virus Integration Site 1 (AAVS1), a genomic "safe harbor" where disruptions are not expected to alter cellular function. In preclinical models of chronic lymphocytic leukemia (CLL) and acute lymphoblastic leukemia (ALL), these cells serve as a negative control to isolate the functional benefits of specific gene disruptions—such as those in CARD8 or SRCAP—on T-cell proliferation, tumor clearance, and long-term survival. While they possess the fundamental anti-tumor activity of the base CAR construct, they lack the enhanced therapeutic properties sought through targeted lentiviral vector integration site (LVIS)-based gene editing.

Other names

AAVS1 control CAR-T cellsAAVS-1 control CAR-T cellsAAVS 1 control CAR-T cellsAAVS1-edited CAR-T cellsAAVS-1-edited CAR-T cellsAAVS 1-edited CAR-T cells