Development of cyclodextrin based materials for gene delivery
Safe and efficient delivery of nucleic acid constructs to target cells has great potential for the treatment of genetic diseases, however, the clinical success of this approach greatly depends on the development of effective delivery vehicles with low toxicity. Herein, a family of cyclodextrin based materials has been developed based on non-covalent interactions and self-assembly for the purpose of nucleic acid delivery. The results suggest that the pendant polymer:cationic CD complexes and cationic polyrotaxanes designed here for pDNA and siRNA delivery are viable candidates for in vivo translation due to their low toxicity, attractive physical characteristics and efficacy. A scalable microfluidic platform has also been developed for the operator independent formulation of nucleic acid nanoparticles with control over size, PDI, zeta potential and disassembly characteristics.
Thompson, Purdue University.
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