Gene therapy: too much splice can spoil the dish.
- Trono D Frontiers-in-Genetics Program and School of Life Sciences, École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland. didier.trono@epfl.ch
- 2012-04-24
Published in:
- The Journal of clinical investigation. - 2012
Alternative Splicing
Genome, Human
Humans
Lentivirus
Lentivirus Infections
Protein Isoforms
Transcriptome
Virus Integration
English
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.
- Language
-
- English
- Open access status
- bronze
- Identifiers
-
- DOI 10.1172/JCI63066
- PMID 22523063
- Persistent URL
- https://sonar.ch/global/documents/96932
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