The DOI matches its Crossref record; 8 of the 8 references it cites were found in a registry or an index.
Hydrophobization and bioconjugation for enhanced siRNA delivery and targeting
Open accessAbstract
RNA interference (RNAi) is an evolutionarily conserved process by which double-stranded small interfering RNA (siRNA) induces sequence-specific, post-transcriptional gene silencing. Unlike other mRNA targeting strategies, RNAi takes advantage of the physiological gene silencing machinery. The potential use of siRNA as therapeutic agents has attracted great attention as a novel approach for treating severe and chronic diseases. RNAi can be achieved by either delivery of chemically synthesized siRNAs or endogenous expression of small hairpin RNA, siRNA, and microRNA (miRNA). However, the relatively high dose of siRNA required for gene silencing limits its therapeutic applications. This review discusses several strategies to improve therapeutic efficacy as well as to abrogate off-target effects and immunostimulation caused by siRNAs. There is an in-depth discussion on various issues related to the (1) mechanisms of RNAi, (2) methods of siRNA production, (3) barriers to RNAi-based therapies, (4) biodistribution, (5) design of siRNA molecules, (6) chemical modification and bioconjugation, (7) complex formation with lipids and polymers, (8) encapsulation into lipid particles, and (9) target specificity for enhanced therapeutic effectiveness.
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Cites
Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
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Expression profiling reveals off-target gene regulation by RNAi
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Rational siRNA design for RNA interference
Reynolds, A., Leake, D., Boese, Q., Scaringe, S., Marshall, W. S., & Khvorova, A. (2004) · Nature Biotechnology · doi:10.1038/nbt936 · Cited by 2,094
Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways
Grimm, D., Streetz, K. L., Jopling, C. L., Storm, T. A., Pandey, K., Davis, C. R., Marion, P., Salazar, F., & Kay, M. A. (2006) · Nature · doi:10.1038/nature04791 · Cited by 1,677
RNAi-mediated gene silencing in non-human primates
Zimmermann, T. S., Lee, A. C. H., Akinc, A., Bramlage, B., Bumcrot, D., Fedoruk, M. N., Harborth, J., Heyes, J. A., Jeffs, L. B., John, M., et al. (2006) · Nature · doi:10.1038/nature04688 · Cited by 1,357
Transcription Start Regions in the Human Genome Are Favored Targets for MLV Integration
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3′ UTR seed matches, but not overall identity, are associated with RNAi off-targets
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Tolerance for mutations and chemical modifications in a siRNA
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Cited by
Silencing disease genes in the laboratory and the clinic
Watts, J. K., & Corey, D. R. (2012) · The Journal of Pathology · doi:10.1002/path.2993 · Cited by 470
Engineering RNA for Targeted siRNA Delivery and Medical Application
Guo, P., Coban, O., Snead, N. M., Trebley, J., Hoeprich, S., Guo, S., & Shu, Y. (2010) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2010.03.008 · Cited by 300
Zhu, L., Perche, F., Wang, T., & Torchilin, V. P. (2014) · Biomaterials · doi:10.1016/j.biomaterials.2014.01.060 · Cited by 220
Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain
Alterman, J. F., Hall, L. M., Coles, A. H., Hassler, M. R., Didiot, M.-C., Chase, K., Abraham, J., Sottosanti, E., Johnson, E., Sapp, E., et al. (2015) · Molecular Therapy - Nucleic Acids · doi:10.1038/mtna.2015.38 · Cited by 158
Targeted Delivery of Small Interfering RNA: Approaching Effective Cancer Therapies
Pirollo, K. F., & Chang, E. H. (2008) · Cancer Research · doi:10.1158/0008-5472.can-07-5810 · Cited by 121
Metadata
- Authors
- De Paula, D., Bentley, M. V. L. B., & Mahato, R. I.
- Venue
- RNA
- Publisher
- Cold Spring Harbor Laboratory
- Published
- 2007-02-28
- Volume
- 13
- Issue
- 4
- Pages
- 431-456
- ISSN
- 1355-8382 (ISSN-L)
- 1469-9001
- OpenAlex
- W2122935177
- Cited by
- 228
- References
- 8
- FWCI
- 8.74
- Type
- journal article
- Language
- en
- Open access
- bronze
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- De Paula, D., Bentley, M. V. L. B., & Mahato, R. I. (2007). Hydrophobization and bioconjugation for enhanced siRNA delivery and targeting. RNA. https://doi.org/10.1261/rna.459807
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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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