Cyclodextrins in drug delivery: applications in gene and combination therapy
Author(s)Haley, Rebecca M.; Gottardi, Riccardo; Langer, Robert S; Mitchell, Michael J.
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Gene therapy is a powerful tool against genetic disorders and cancer, targeting the source of the disease rather than just treating the symptoms. While much of the initial success of gene delivery relied on viral vectors, non-viral vectors are emerging as promising gene delivery systems for efficacious treatment with decreased toxicity concerns. However, the delivery of genetic material is still challenging, and there is a need for vectors with enhanced targeting, reduced toxicity, and controlled release. In this article, we highlight current work in gene therapy which utilizes the cyclic oligosaccharide molecule cyclodextrin (CD). With a number of unique abilities, such as hosting small molecule drugs, acting as a linker or modular component, reducing immunogenicity, and disrupting membranes, CD is a valuable constituent in many delivery systems. These carriers also demonstrate great promise in combination therapies, due to the ease of assembling macromolecular structures and wide variety of chemical derivatives, which allow for customizable delivery systems and co-delivery of therapeutics. The use of combination and personalized therapies can result in improved patient health—modular systems, such as those which incorporate CD, are more conducive to these therapy types.
DepartmentMassachusetts Institute of Technology. Department of Chemical Engineering; Massachusetts Institute of Technology. Department of Biological Engineering; Koch Institute for Integrative Cancer Research at MIT
Drug Delivery and Translational Research
Springer Science and Business Media LLC
Haley, Rebecca M. et al. "Cyclodextrins in drug delivery: applications in gene and combination therapy." Drug Delivery and Translational Research (June 2020): 661–677 © 2020 Controlled Release Society
Author's final manuscript