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

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 c...

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Main Authors: Chiara F. Magnani, Sarah Tettamanti, Gaia Alberti, Ilaria Pisani, Andrea Biondi, Marta Serafini, Giuseppe Gaipa
Format: Article
Language:English
Published: MDPI AG 2020-05-01
Series:Cells
Subjects:
Online Access:https://www.mdpi.com/2073-4409/9/6/1337
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author Chiara F. Magnani
Sarah Tettamanti
Gaia Alberti
Ilaria Pisani
Andrea Biondi
Marta Serafini
Giuseppe Gaipa
author_facet Chiara F. Magnani
Sarah Tettamanti
Gaia Alberti
Ilaria Pisani
Andrea Biondi
Marta Serafini
Giuseppe Gaipa
author_sort Chiara F. Magnani
collection DOAJ
description 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.
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spelling doaj.art-c3c75952a1914ec19fe9141e59331da42023-11-20T01:54:36ZengMDPI AGCells2073-44092020-05-0196133710.3390/cells9061337Transposon-Based CAR T Cells in Acute Leukemias: Where Are We Going?Chiara F. Magnani0Sarah Tettamanti1Gaia Alberti2Ilaria Pisani3Andrea Biondi4Marta Serafini5Giuseppe Gaipa6Centro Ricerca M. Tettamanti, Clinica Pediatrica, Università di Milano-Bicocca, Ospedale San Gerardo, 20900 Monza, ItalyCentro Ricerca M. Tettamanti, Clinica Pediatrica, Università di Milano-Bicocca, Ospedale San Gerardo, 20900 Monza, ItalyCentro Ricerca M. Tettamanti, Clinica Pediatrica, Università di Milano-Bicocca, Ospedale San Gerardo, 20900 Monza, ItalyCentro Ricerca M. Tettamanti, Clinica Pediatrica, Università di Milano-Bicocca, Ospedale San Gerardo, 20900 Monza, ItalyCentro Ricerca M. Tettamanti, Clinica Pediatrica, Università di Milano-Bicocca, Ospedale San Gerardo, 20900 Monza, ItalyCentro Ricerca M. Tettamanti, Clinica Pediatrica, Università di Milano-Bicocca, Ospedale San Gerardo, 20900 Monza, ItalyCentro Ricerca M. Tettamanti, Clinica Pediatrica, Università di Milano-Bicocca, Ospedale San Gerardo, 20900 Monza, ItalyChimeric 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.https://www.mdpi.com/2073-4409/9/6/1337CAR T-cellssleeping beautytransposonacute leukemiagene transferimmunotherapy
spellingShingle Chiara F. Magnani
Sarah Tettamanti
Gaia Alberti
Ilaria Pisani
Andrea Biondi
Marta Serafini
Giuseppe Gaipa
Transposon-Based CAR T Cells in Acute Leukemias: Where Are We Going?
Cells
CAR T-cells
sleeping beauty
transposon
acute leukemia
gene transfer
immunotherapy
title Transposon-Based CAR T Cells in Acute Leukemias: Where Are We Going?
title_full Transposon-Based CAR T Cells in Acute Leukemias: Where Are We Going?
title_fullStr Transposon-Based CAR T Cells in Acute Leukemias: Where Are We Going?
title_full_unstemmed Transposon-Based CAR T Cells in Acute Leukemias: Where Are We Going?
title_short Transposon-Based CAR T Cells in Acute Leukemias: Where Are We Going?
title_sort transposon based car t cells in acute leukemias where are we going
topic CAR T-cells
sleeping beauty
transposon
acute leukemia
gene transfer
immunotherapy
url https://www.mdpi.com/2073-4409/9/6/1337
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AT ilariapisani transposonbasedcartcellsinacuteleukemiaswherearewegoing
AT andreabiondi transposonbasedcartcellsinacuteleukemiaswherearewegoing
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