Gene therapy: too much splice can spoil the dish

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.


Published in:
The Journal of clinical investigation, 122, 5, 1600-1602
Year:
2012
ISSN:
1558-8238
Keywords:
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 Record created 2012-04-27, last modified 2018-01-28

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