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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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
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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
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Transient and stable vector transfection: Pitfalls, off-target effects, artifacts
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Targeted Genome Modifications Using Integrase-deficient Lentiviral Vectors
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Systemic modified messenger RNA for replacement therapy in alpha 1-antitrypsin deficiency
Karadagi, A., Cavedon, A. G., Zemack, H., Nowak, G., Eybye, M. E., Zhu, X., Guadagnin, E., White, R. A., Rice, L. M., Frassetto, A. L., et al. (2020) · Scientific Reports · doi:10.1038/s41598-020-64017-0 · Cited by 54
Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration
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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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