The DOI matches its Crossref record; 3 of the 3 references it cites were found in a registry or an index.
The First Targeted Delivery of siRNA in Humans via a Self-Assembling, Cyclodextrin Polymer-Based Nanoparticle: From Concept to Clinic
Abstract
Experimental therapeutics developed to exploit RNA interference (RNAi) are now in clinical studies. Here, the translation from concept to clinic for the first experimental therapeutic to provide targeted delivery of synthetic, small interfering RNA (siRNA) in humans is described. This targeted, nanoparticle formulation of siRNA, denoted as CALAA-01, consists of a cyclodextrin-containing polymer (CDP), a polythethylene glycol (PEG) steric stabilization agent, and human transferrin (Tf) as a targeting ligand for binding to transferrin receptors (TfR) that are typically upregulated on cancer cells. The four component formulation is self-assembled into nanoparticles in the pharmacy and administered intravenously (iv) to patients. The designed features of this experimental therapeutic are described, and their functions illustrated.
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Citations
Cites
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-citationThe proton sponge: a trick to enter cells the viruses did not exploit
Behr, J.-P. (1997) · CHIMIA · doi:10.2533/chimia.1997.34 · Cited by 933
Non-viral gene delivery systems
Davis, M. E. (2002) · Current Opinion in Biotechnology · doi:10.1016/s0958-1669(02)00294-x · Cited by 419
Self-citation
Cited by
Cancer nanomedicine: progress, challenges and opportunities
Shi, J., Kantoff, P. W., Wooster, R., & Farokhzad, O. C. (2017) · Nature Reviews Cancer · doi:10.1038/nrc.2016.108 · Cited by 5,873
PEGylation as a strategy for improving nanoparticle-based drug and gene delivery
Suk, J. S., Xu, Q., Kim, N., Hanes, J., & Ensign, L. M. (2016) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2015.09.012 · Cited by 4,523
Strategies in the design of nanoparticles for therapeutic applications
Petros, R. A., & DeSimone, J. M. (2010) · Nature Reviews Drug Discovery · doi:10.1038/nrd2591 · Cited by 3,651
Non-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
Kamaly, N., Yameen, B., Wu, J., & Farokhzad, O. C. (2016) · Chemical Reviews · doi:10.1021/acs.chemrev.5b00346 · Cited by 3,070
Metadata
- Authors
- Davis, M. E.
- Venue
- Molecular Pharmaceutics
- Publisher
- American Chemical Society (ACS)
- Published
- 2009-04-03
- Volume
- 6
- Issue
- 3
- Pages
- 659-668
- ISSN
- 1543-8384 (ISSN-L)
- 1543-8392
- OpenAlex
- W2108882170
- Cited by
- 977
- References
- 3
- FWCI
- 28.45
- Type
- journal article
- Language
- en
- Open access
- closed
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