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GMR CAR-T cells (E21R) is an experimental adoptive cell therapy consisting of autologous T cells engineered to express a chimeric antigen receptor (CAR) targeting the granulocyte-macrophage colony-stimulating factor receptor (GMR) complex, specifically the CD116/CD131 heterodimer. Developed by researchers at Shinshu University, the therapy utilizes a non-viral piggyBac transposon system for gene transfer. The CAR construct features a mutated human GM-CSF ligand as the antigen-binding domain, specifically incorporating a glutamate-to-arginine substitution at residue 21 (E21R) and a G4S spacer to enhance binding affinity and anti-tumor activity while minimizing off-target effects. It is primarily being investigated for the treatment of relapsed or refractory acute myeloid leukemia (AML) and juvenile myelomonocytic leukemia (JMML), where CD116 is frequently overexpressed.
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