The DOI matches its Crossref record; 52 of the 52 references it cites were found in a registry or an index.
Sequence-Specific Knockdown of EWS-FLI1 by Targeted, Nonviral Delivery of Small Interfering RNA Inhibits Tumor Growth in a Murine Model of Metastatic Ewing's Sarcoma
Abstract
The development of effective, systemic therapies for metastatic cancer is highly desired. We show here that the systemic delivery of sequence-specific small interfering RNA (siRNA) against the EWS-FLI1 gene product by a targeted, nonviral delivery system dramatically inhibits tumor growth in a murine model of metastatic Ewing's sarcoma. The nonviral delivery system uses a cyclodextrin-containing polycation to bind and protect siRNA and transferrin as a targeting ligand for delivery to transferrin receptor–expressing tumor cells. Removal of the targeting ligand or the use of a control siRNA sequence eliminates the antitumor effects. Additionally, no abnormalities in interleukin-12 and IFN-α, liver and kidney function tests, complete blood counts, or pathology of major organs are observed from long-term, low-pressure, low-volume tail-vein administrations. These data provide strong evidence for the safety and efficacy of this targeted, nonviral siRNA delivery system.
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Cites
Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
Soutschek, J., Akinc, A., Bramlage, B., Charisse, K., Constien, R., Donoghue, M., Elbashir, S., Geick, A., Hadwiger, P., Harborth, J., et al. (2004) · Nature · doi:10.1038/nature03121 · Cited by 2,183
Rubinson, D. A., Dillon, C. P., Kwiatkowski, A. V., Sievers, C., Yang, L., Kopinja, J., Rooney, D. L., Zhang, M., Ihrig, M. M., McManus, M. T., et al. (2003) · Nature Genetics · doi:10.1038/ng1117 · Cited by 1,501
Activation of the interferon system by short-interfering RNAs
Sledz, C. A., Holko, M., de Veer, M. J., Silverman, R. H., & Williams, B. R. G. (2003) · Nature Cell Biology · doi:10.1038/ncb1038 · Cited by 1,400
Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA
Judge, A. D., Sood, V., Shaw, J. R., Fang, D., McClintock, K., & MacLachlan, I. (2005) · Nature Biotechnology · doi:10.1038/nbt1081 · Cited by 1,250
Hornung, V., Guenthner-Biller, M., Bourquin, C., Ablasser, A., Schlee, M., Uematsu, S., Noronha, A., Manoharan, M., Akira, S., de Fougerolles, A., et al. (2005) · Nature Medicine · doi:10.1038/nm1191 · Cited by 1,232
RNA interference targeting Fas protects mice from fulminant hepatitis
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Delattre, O., Zucman, J., Melot, T., Garau, X. S., Zucker, J.-M., Lenoir, G. M., Ambros, P. F., Sheer, D., Turc-Carel, C., Triche, T. J., et al. (1994) · New England Journal of Medicine · doi:10.1056/nejm199408043310503 · Cited by 1,107
Self-citationRNA interference in adult mice
McCaffrey, A. P., Meuse, L., Pham, T.-T. T., Conklin, D. S., Hannon, G. J., & Kay, M. A. (2002) · Nature · doi:10.1038/418038a · Cited by 1,059
Induction of an interferon response by RNAi vectors in mammalian cells
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Cited by
Nanoparticle therapeutics: an emerging treatment modality for cancer
Davis, M. E., Chen, Z., & Shin, D. M. (2008) · Nature Reviews Drug Discovery · doi:10.1038/nrd2614 · Cited by 4,233
Self-citationNon-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
Knocking down barriers: advances in siRNA delivery
Whitehead, K. A., Langer, R., & Anderson, D. G. (2009) · Nature Reviews Drug Discovery · doi:10.1038/nrd2742 · Cited by 3,115
Nonviral vectors for gene delivery
Mintzer, M. A., & Simanek, E. E. (2009) · Chemical Reviews · doi:10.1021/cr800409e · Cited by 2,366
Delivery materials for siRNA therapeutics
Kanasty, R., Dorkin, J. R., Vegas, A., & Anderson, D. (2013) · Nature Materials · doi:10.1038/nmat3765 · Cited by 1,800
Metadata
- Authors
- Hu-Lieskovan, S., Heidel, J. D., Bartlett, D. W., Davis, M. E., & Triche, T. J.
- Venue
- Cancer Research
- Published
- 2005-10-01
- Volume
- 65
- Issue
- 19
- Pages
- 8984-8992
- ISSN
- 0008-5472 (ISSN-L)
- 1538-7445
- OpenAlex
- W2015511149
- Cited by
- 567
- References
- 52
- FWCI
- 14.29
- Type
- journal article
- Language
- en
- Open access
- closed
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- CoChat. (2026). Citation check: Sequence-Specific Knockdown of EWS-FLI1 by Targeted, Nonviral Delivery of Small Interfering RNA Inhibits Tumor Growth in a Murine Model of Metastatic Ewing's Sarcoma. https://app.cochat.ai/papers/10.1158/0008-5472.can-05-0565
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- Hu-Lieskovan, S., Heidel, J. D., Bartlett, D. W., Davis, M. E., & Triche, T. J. (2005). Sequence-Specific Knockdown of EWS-FLI1 by Targeted, Nonviral Delivery of Small Interfering RNA Inhibits Tumor Growth in a Murine Model of Metastatic Ewing's Sarcoma. Cancer Research. https://doi.org/10.1158/0008-5472.can-05-0565
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