TY - JOUR T1 - Establishment of a simple and efficient platform for car-t cell generation and expansion: from lentiviral production to in vivo studies JO - Hematology, Transfusion and Cell Therapy T2 - AU - Picanço-Castro,Virgínia AU - Moço,Pablo Diego AU - Mizukami,Amanda AU - Vaz,Leticia Delfini AU - de Souza Fernandes Pereira,Marcelo AU - Silvestre,Renata Nacasaki AU - de Azevedo,Júlia Teixeira Cottas AU - de Sousa Bomfim,Aline AU - de Abreu Neto,Mario Soares AU - Malmegrim,Kelen Cristina Ribeiro AU - Swiech,Kamilla AU - Covas,Dimas Tadeu SN - 25311379 M3 - 10.1016/j.htct.2019.06.007 DO - 10.1016/j.htct.2019.06.007 UR - http://www.htct.com.br/en-establishment-simple-efficient-platform-for-articulo-S253113791930135X AB - IntroductionAdoptive transfer of T cells expressing a CD19-specific chimeric antigen receptor (CAR) has shown impressive response rates for the treatment of CD19 + B-cell malignancies in numerous clinical trials. The CAR molecule, which recognizes cell-surface tumor-associated antigen independently of human leukocyte antigen (HLA), is composed by one or more signaling molecules to activate genetically modified T cells for killing, proliferation, and cytokine production. ObjectivesIn order to make this treatment available for a larger number of patients, we developed a simple and efficient platform to generate and expand CAR-T cells. MethodsOur approach is based on a lentiviral vector composed by a second-generation CAR that signals through a 41BB and CD3-ζ endodomain. ConclusionsIn this work, we show a high-level production of the lentiviral vector, which was successfully used to generate CAR-T cells. The CAR-T cells produced were highly cytotoxic and specific against CD19+ cells in vitro and in vivo, being able to fully control disease progression in a xenograft B-cell lymphoma mouse model. Our work demonstrates the feasibility of producing CAR-T cells in an academic context and can serve as a paradigm for similar institutions. Nevertheless, the results presented may contribute favoring the translation of the research to the clinical practice. ER -