Lentiviral vectors for inducible, transactivator-free advanced therapy medicinal products: Application to CAR-T cells.

ABSTRACT: Controlling transgene expression through an externally administered inductor is envisioned as a potent strategy to improve safety and efficacy of gene therapy approaches. Generally, inducible ON systems require a chimeric transcription factor (transactivator) that becomes activated by an inductor, which is not optimal for clinical translation due to their toxicity. We generated previously the first all-in-one, transactivator-free, doxycycline (Dox)-responsive (Lent-On-Plus or LOP) lentiviral vectors (LVs) able to control transgene expression in human stem cells. Here, we have generated new versions of the LOP LVs and have analyzed their applicability for the generation of inducible advanced therapy medicinal products (ATMPs) with special focus on primary human T cells. We have shown that,

SUBMITTER: Tristan-Manzano M 

PROVIDER: S-EPMC10141506 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature