期刊
MOLECULAR THERAPY
卷 18, 期 4, 页码 674-683出版社
CELL PRESS
DOI: 10.1038/mt.2010.2
关键词
-
资金
- NIH [P01HD32652, R01DK082516, R01CA113636, RO1CA124782, R01CA120956]
The stable introduction of therapeutic transgenes into human cells can be accomplished using viral and non-viral approaches. Transduction with clinical-grade recombinant viruses offers the potential of efficient gene transfer into primary cells and has a record of therapeutic successes. However, widespread application for gene therapy using viruses can be limited by their initially high cost of manufacture at a limited number of production facilities as well as a propensity for nonrandom patterns of integration. The ex vivo application of transposon-mediated gene transfer now offers an alternative to the use of viral vectors. Clinical-grade DNA plasmids can be prepared at much reduced cost and with lower immunogenicity, and the integration efficiency can be improved by the transient coexpression of a hyperactive transposase. This has facilitated the design of human trials using the Sleeping Beauty (SB) transposon system to introduce a chimeric antigen receptor (CAR) to redirect the specificity of human T cells. This review examines the rationale and safety implications of application of the SB system to genetically modify T cells to be manufactured in compliance with current good manufacturing practice (cGMP) for phase I/II trials.
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