Transposon-Based CAR T Cells in Acute Leukemias: Where are We Going?

Cells. 2020 May 27;9(6):1337. doi: 10.3390/cells9061337.


Chimeric Antigen Receptor (CAR) T-cell therapy has become a new therapeutic reality for refractory and relapsed leukemia patients and is also emerging as a potential therapeutic option in solid tumors. Viral vector-based CAR T-cells initially drove these successful efforts; however, high costs and cumbersome manufacturing processes have limited the widespread clinical implementation of CAR T-cell therapy. Here we will discuss the state of the art of the transposon-based gene transfer and its application in CAR T immunotherapy, specifically focusing on the Sleeping Beauty (SB) transposon system, as a valid cost-effective and safe option as compared to the viral vector-based systems. A general overview of SB transposon system applications will be provided, with an update of major developments, current clinical trials achievements and future perspectives exploiting SB for CAR T-cell engineering. After the first clinical successes achieved in the context of B-cell neoplasms, we are now facing a new era and it is paramount to advance gene transfer technology to fully exploit the potential of CAR T-cells towards next-generation immunotherapy.

Keywords: CAR T-cells; acute leukemia; gene transfer; immunotherapy; sleeping beauty; transposon.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Acute Disease
  • Clinical Trials as Topic
  • DNA Transposable Elements / genetics*
  • Humans
  • Leukemia / genetics
  • Leukemia / immunology*
  • Receptors, Chimeric Antigen / immunology*
  • T-Lymphocytes / immunology*


  • DNA Transposable Elements
  • Receptors, Chimeric Antigen