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Cyclodextrin/Dendrimer conjugates as DNA and oligonucleotide carriers
Hidetoshi Arima, Keiichi Motoyama, Taishi Higashi1
1Graduate School of Pharmaceutical Sciences, Kumamoto University, 5-1 Oe-honmachi, Chuo-ku, Kumamoto 862-0973, Japan. arimah@gpo.kumamoto-u.ac.jp.
Current Topics in Medicinal Chemistry
|December 21, 2013
Summary
Cyclodextrin (CyD)-grafted polymers, known as CDEs, show promise as effective gene and oligonucleotide carriers. Specific conjugates, like dendrimer-grafted α-CyD and glucuronosylglucosyl-β-CyD, demonstrate high transfer activity with low toxicity.
Area of Science:
- Biomaterials Science
- Nanotechnology
- Gene Delivery
Background:
- Gene and oligonucleotide therapy requires efficient and safe delivery systems.
- Cyclodextrin (CyD)-grafted polymers and supramolecules are emerging as promising carriers.
- Polyamidoamine starburst dendrimers conjugated with CyDs (CDEs) have shown potential in this field.
Purpose of the Study:
- To review the potential of various cyclodextrin-dendrimer conjugates (CDEs) as gene and oligonucleotide transfer carriers.
- To highlight CDEs with high gene transfer activity and low cytotoxicity.
- To discuss modifications for targeted gene delivery and sustained release.
Main Methods:
- Synthesis and characterization of various cyclodextrin-dendrimer conjugates (CDEs).
- Evaluation of gene and oligonucleotide transfer activity in vitro.
- Assessment of cytotoxicity of the developed CDEs.
- Modification of CDEs with functional molecules for targeted delivery and sustained release.
Main Results:
- Dendrimer (G2 or G3) conjugates with α-cyclodextrin (α-CDE) exhibited high gene and oligonucleotide transfer activity with negligible cytotoxicity.
- α-CDEs modified with functional molecules (mannose, folate-PEG, etc.) were developed for tissue- or cell-specific gene transfer.
- Polypseudorotaxane-appended α-CDEs demonstrated sustained release of DNA and siRNA.
- Glucuronosylglucosyl-β-cyclodextrin (GUG-β-CDE (G2)) showed superior gene transfer activity compared to α-CDE (G2) and β-CDE (G2).
Conclusions:
- Cyclodextrin-dendrimer conjugates (CDEs) represent a versatile platform for gene and oligonucleotide delivery.
- Specific CDE structures, including modified and appended versions, offer enhanced efficacy, targeting, and release profiles.
- CDEs hold significant potential for advancing gene therapy and oligonucleotide-based treatments.
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