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Gene therapy: too much splice can spoil the dish.

Type of publication Peer-reviewed
Publikationsform Original article (peer-reviewed)
Publication date 2012
Author Trono Didier,
Project Innate defenses against retroelements
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Original article (peer-reviewed)

Journal The Journal of clinical investigation
Volume (Issue) 122(5)
Page(s) 1600 - 2
Title of proceedings The Journal of clinical investigation
DOI 10.1172/JCI63066


The use of integrating vectors for gene therapy - required for stable correction of gene expression - carries the risk of insertional mutagenesis, which can lead to activation of a tumorigenic program. In this issue of the JCI, Moiani et al. and Cesana et al. investigate how viral vectors can induce aberrant splicing, resulting in chimeric cellular-viral transcripts. The finding that this is a general phenomenon is concerning, but some of their results do suggest approaches for the development of safeguards in gene therapy vector design.