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A Nonviral Approach to Generate Transient Chimeric Antigen Receptor T Cells Using mRNA for Cancer Immunotherapy
Published on: February 21, 2025
Promises and potential pitfalls of in vivo CAR gene therapy
Dimitrios L Wagner1,2, Magdi Elsallab3,4, Marcela V Maus3,4
1Center for Cell and Gene Therapy and Department of Molecular and Cellular Biology, Baylor College of Medicine, Houston, TX.
Abstract:
Genetically modified T cells expressing chimeric antigen receptors (CARs) manufactured ex vivo have demonstrated high efficacy in patients with lymphoid hematologic malignancies, but their logistic complexity limits patient access. In vivo approaches generate the CAR-expressing T cells within the body but may have their own challenges. Recent clinical reports provide proof of concept for the feasibility and therapeutic potential of in vivo CAR gene delivery. We summarize clinical evidence and discuss the critical technical, biological, and safety considerations of this approach.
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