The DOI matches its Crossref record; 7 of the 7 references it cites were found in a registry or an index.
Chance or necessity? Insertional Mutagenesis in Gene Therapy and Its Consequences
Open accessAbstract
Recently, unusual forms of leukemias have developed as complications following retroviral transfer of potentially therapeutic genes into hematopoietic cells. A crucial component in the pathogenesis of these complications was the upregulation of a cellular proto-oncogene by random insertion of the retroviral gene transfer vector. These findings have great implications for the genetic manipulation of somatic stem cells in medicine. This review discusses the extent to which the random oncogene activation may have required disease-specific stimuli of the transgene and the hematopoietic milieu to become leukemogenic. Based on these considerations, we propose approaches to risk prediction and prevention.
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Cites
HIV-1 Integration in the Human Genome Favors Active Genes and Local Hotspots
Schröder, A. R. W., Shinn, P., Chen, H., Berry, C., Ecker, J. R., & Bushman, F. (2002) · Cell · doi:10.1016/s0092-8674(02)00864-4 · Cited by 1,832
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
Evidence for a mitotic clock in human hematopoietic stem cells: loss of telomeric DNA with age.
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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
AAV serotype 2 vectors preferentially integrate into active genes in mice
Nakai, H., Montini, E., Fuess, S., Storm, T. A., Grompe, M., & Kay, M. A. (2003) · Nature Genetics · doi:10.1038/ng1179 · Cited by 392
Allsopp, R. C., Morin, G. B., DePinho, R., Harley, C. B., & Weissman, I. L. (2003) · Blood · doi:10.1182/blood-2002-07-2334 · Cited by 324
Chang, S., Khoo, C. M., Naylor, M. L., Maser, R. S., & DePinho, R. A. (2003) · Genes & Development · doi:10.1101/gad.1029903 · Cited by 183
Cited by
Ott, M. G., Schmidt, M., Schwarzwaelder, K., Stein, S., Siler, U., Koehl, U., Glimm, H., Kühlcke, K., Schilz, A., Kunkel, H., et al. (2006) · Nature Medicine · doi:10.1038/nm1393 · Cited by 1,051
Self-citationLombardo, A., Genovese, P., Beausejour, C. M., Colleoni, S., Lee, Y.-L., Kim, K. A., Ando, D., Urnov, F. D., Galli, C., Gregory, P. D., et al. (2007) · Nature Biotechnology · doi:10.1038/nbt1353 · Cited by 845
Genome-editing Technologies for Gene and Cell Therapy
Maeder, M. L., & Gersbach, C. A. (2016) · Molecular Therapy · doi:10.1038/mt.2016.10 · Cited by 719
Genomic Instability of iPSCs: Challenges Towards Their Clinical Applications
Yoshihara, M., Hayashizaki, Y., & Murakawa, Y. (2017) · Stem Cell Reviews and Reports · doi:10.1007/s12015-016-9680-6 · Cited by 283
Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis
Robinson, E., MacDonald, K. D., Slaughter, K., McKinney, M., Patel, S., Sun, C., & Sahay, G. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.05.014 · Cited by 272
Metadata
- Authors
- Baum, C., von Kalle, C., Staal, F. J. T., Li, Z., Fehse, B., Schmidt, M., Weerkamp, F., Karlsson, S., Wagemaker, G., & Williams, D. A.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2004-01
- Volume
- 9
- Issue
- 1
- Pages
- 5-13
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W2151137440
- Cited by
- 225
- References
- 7
- FWCI
- 16.84
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- Baum, C., von Kalle, C., Staal, F. J. T., Li, Z., Fehse, B., Schmidt, M., Weerkamp, F., Karlsson, S., Wagemaker, G., & Williams, D. A. (2004). Chance or necessity? Insertional Mutagenesis in Gene Therapy and Its Consequences. Molecular Therapy. https://doi.org/10.1016/j.ymthe.2003.10.013
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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