The DOI matches its Crossref record; 7 of the 7 references it cites were found in a registry or an index.
Mutagenesis and Oncogenesis by Chromosomal Insertion of Gene Transfer Vectors
Abstract
Increasing evidence reveals that random insertion of gene transfer vectors into the genome of repopulating hematopoietic cells may alter their fate in vivo. Although most insertional mutations are expected to have few if any consequences for cellular survival, clonal dominance caused by retroviral vector insertions in (or in the vicinity of) proto-oncogenes or other signaling genes has been described for both normal and malignant hematopoiesis. Important insights into these side effects were initially obtained in murine models. Results from ongoing clinical studies have revealed that similar adverse events may also occur in human gene therapy. However, it remains unknown to what extent the outcome of insertional mutagenesis induced by gene vectors is related to (1) the architecture and type of vector used, (2) intrinsic properties of the target cell, and (3) extrinsic and potentially disease-specific factors influencing clonal competition in vivo. This review discusses reports addressing these questions, underlining the need for models that demonstrate and quantify the functional consequences of insertional mutagenesis. Improving vector design appears to be the most straightforward approach to increase safety, provided all relevant cofactors are considered.
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Cites
HIV-1 Integration in the Human Genome Favors Active Genes and Local Hotspots
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Transcription Start Regions in the Human Genome Are Favored Targets for MLV Integration
Wu, X., Li, Y., Crise, B., & Burgess, S. M. (2003) · Science · doi:10.1126/science.1083413 · Cited by 1,305
Rules for Making Human Tumor Cells
Hahn, W. C., & Weinberg, R. A. (2002) · New England Journal of Medicine · doi:10.1056/nejmra021902 · Cited by 951
Correction of sickle cell disease in transgenic mouse models by gene therapy
Pawliuk, R., Westerman, K. A., Fabry, M. E., Payen, E., Tighe, R., Bouhassira, E. E., Acharya, S. A., Ellis, J., London, I. M., Eaves, C. J., et al. (2001) · Science · doi:10.1126/science.1065806 · Cited by 579
AAV serotype 2 vectors preferentially integrate into active genes in mice
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Large‐scale molecular characterization of adeno‐associated virus vector integration in mouse liver
Nakai, H., Wu, X., Fuess, S., Storm, T. A., Munroe, D., Montini, E., Burgess, S. M., Grompe, M., & Kay, M. A. (2005) · Journal of Virology · doi:10.1128/jvi.79.6.3606-3614.2005 · Cited by 190
Custom zinc-finger nucleases for use in human cells
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Non-viral vectors for gene-based therapy
Yin, H., Kanasty, R. L., Eltoukhy, A. A., Vegas, A. J., Dorkin, J. R., & Anderson, D. G. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3763 · Cited by 3,123
siRNA versus miRNA as therapeutics for gene silencing
Lam, J. K. W., Chow, M. Y. T., Zhang, Y., & Leung, S. W. S. (2015) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2015.23 · Cited by 1,081
mRNA vaccine delivery using lipid nanoparticles
Reichmuth, A. M., Oberli, M. A., Jaklenec, A., Langer, R., & Blankschtein, D. (2016) · Therapeutic Delivery · doi:10.4155/tde-2016-0006 · Cited by 624
Delivery technologies for genome editing
Yin, H., Kauffman, K. J., & Anderson, D. G. (2017) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2016.280 · Cited by 597
Current applications and future perspective of CRISPR/Cas9 gene editing in cancer
Wang, S.-W., Gao, C., Zheng, Y.-M., Yi, L., Lu, J.-C., Huang, X.-Y., Cai, J.-B., Zhang, P.-F., Cui, Y.-H., & Ke, A.-W. (2022) · Molecular Cancer · doi:10.1186/s12943-022-01518-8 · Cited by 478
Metadata
- Authors
- Baum, C., Kustikova, O., Modlich, U., Li, Z., & Fehse, B.
- Venue
- Human Gene Therapy
- Publisher
- SAGE Publications
- Published
- 2006-03
- Volume
- 17
- Issue
- 3
- Pages
- 253-263
- ISSN
- 1043-0342 (ISSN-L)
- 1557-7422
- OpenAlex
- W2045015558
- Cited by
- 360
- References
- 7
- FWCI
- 13.08
- Type
- journal article
- Language
- en
- Open access
- closed
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