DIALing-up the preclinical characterization of gene-modified adoptive cellular immunotherapies.

ABSTRACT: The preclinical characterization of gene modified adoptive cellular immunotherapy candidates for clinical development often requires the use of mouse models. Gene-modified lymphocytes (GML) incorporating chimeric antigen receptors (CAR) and T-cell receptors (TCR) into immune effector cells require in vivo characterization of biological activity, mechanism of action, and preclinical safety. Typically, this characterization involves the assessment of dose-dependent, on-target, on-tumor activity in severely immunocompromised mice. While suitable for the purpose of evaluating T cell-expressed transgene function in a living host, this approach falls short in translating cellular therapy efficacy, safety, and persistence from preclinical models to humans. To comprehensively characterize c

SUBMITTER: Giardino Torchia ML 

PROVIDER: S-EPMC10713823 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature