The DOI matches its Crossref record; 255 of the 255 references it cites were found in a registry or an index.
Rekindling RNAi Therapy: Materials Design Requirements for In Vivo siRNA Delivery
Open accessAbstract
With the recent FDA approval of the first siRNA‐derived therapeutic, RNA interference (RNAi)‐mediated gene therapy is undergoing a transition from research to the clinical space. The primary obstacle to realization of RNAi therapy has been the delivery of oligonucleotide payloads. Therefore, the main aims is to identify and describe key design features needed for nanoscale vehicles to achieve effective delivery of siRNA‐mediated gene silencing agents in vivo. The problem is broken into three elements: 1) protection of siRNA from degradation and clearance; 2) selective homing to target cell types; and 3) cytoplasmic release of the siRNA payload by escaping or bypassing endocytic uptake. The in vitro and in vivo gene silencing efficiency values that have been reported in publications over the past decade are quantitatively summarized by material type (lipid, polymer, metal, mesoporous silica, and porous silicon), and the overall trends in research publication and in clinical translation are discussed to reflect on the direction of the RNAi therapeutics field.
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1 retracted
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Cited by
Therapeutic siRNA: state of the art
Hu, B., Zhong, L., Weng, Y., Peng, L., Huang, Y., Zhao, Y., & Liang, X.-J. (2020) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-020-0207-x · Cited by 1,586
Non-viral vectors for RNA delivery
Yan, Y., Liu, X.-Y., Lu, A., Wang, X.-Y., Jiang, L.-X., & Wang, J.-C. (2022) · Journal of Controlled Release · doi:10.1016/j.jconrel.2022.01.008 · Cited by 332
Therapeutic reversal of Huntington’s disease by in vivo self-assembled siRNAs
Zhang, L., Wu, T., Shan, Y., Li, G., Ni, X., Chen, X., Hu, X., Lin, L., Li, Y., Guan, Y., et al. (2021) · Brain · doi:10.1093/brain/awab354 · Cited by 97
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Metadata
- Authors
- Kim, B., Park, J., & Sailor, M. J.
- Venue
- Advanced Materials
- Publisher
- Wiley
- Published
- 2019-09-30
- Volume
- 31
- Issue
- 49
- Pages
- e1903637-e1903637
- ISSN
- 0935-9648 (ISSN-L)
- 1521-4095
- OpenAlex
- W2977584490
- Cited by
- 298
- References
- 255
- FWCI
- 9.67
- Type
- journal article
- Language
- en
- Open access
- hybrid
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