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CD171-specific chimeric antigen receptor (CAR) T-cell therapy is an autologous cellular immunotherapy developed by Seattle Children's Hospital for the treatment of recurrent or refractory neuroblastoma and ganglioneuroblastoma. The therapy targets CD171 (also known as L1-CAM), a cell adhesion molecule that is abundantly expressed on neuroblastoma tumor cells. The CAR construct utilizes the CE7 monoclonal antibody as the recognition domain, which specifically binds to a glycosylation-dependent, tumor-specific epitope on CD171. The therapy involves lentivirally transducing autologous T cells to express either second-generation CARs (containing a 4-1BB costimulatory domain) or third-generation CARs (containing both CD28 and 4-1BB costimulatory domains). Additionally, the construct incorporates a truncated human epidermal growth factor receptor (EGFRt) as a selection marker and potential safety switch. The therapy is typically administered as a defined 1:1 ratio of CD4+ and CD8+ T cells.
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