Cyclodextrins in drug delivery: applications in gene and combination therapy

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 efficaci...

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Main Authors: Haley, Rebecca M., Gottardi, Riccardo, Langer, Robert S, Mitchell, Michael J.
Other Authors: Massachusetts Institute of Technology. Department of Chemical Engineering
Format: Article
Language:English
Published: Springer Science and Business Media LLC 2020
Online Access:https://hdl.handle.net/1721.1/128393
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author Haley, Rebecca M.
Gottardi, Riccardo
Langer, Robert S
Mitchell, Michael J.
author2 Massachusetts Institute of Technology. Department of Chemical Engineering
author_facet Massachusetts Institute of Technology. Department of Chemical Engineering
Haley, Rebecca M.
Gottardi, Riccardo
Langer, Robert S
Mitchell, Michael J.
author_sort Haley, Rebecca M.
collection MIT
description 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.
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spelling mit-1721.1/1283932022-09-29T11:32:25Z Cyclodextrins in drug delivery: applications in gene and combination therapy Haley, Rebecca M. Gottardi, Riccardo Langer, Robert S Mitchell, Michael J. Massachusetts Institute of Technology. Department of Chemical Engineering Massachusetts Institute of Technology. Department of Biological Engineering Koch Institute for Integrative Cancer Research at MIT 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. 2020-11-06T21:06:09Z 2020-11-06T21:06:09Z 2020-02 2020-09-24T21:47:26Z Article http://purl.org/eprint/type/JournalArticle 2190-393X 2190-3948 https://hdl.handle.net/1721.1/128393 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 en http://dx.doi.org/10.1007/s13346-020-00724-5 Drug Delivery and Translational Research Creative Commons Attribution-Noncommercial-Share Alike http://creativecommons.org/licenses/by-nc-sa/4.0/ Controlled Release Society application/pdf Springer Science and Business Media LLC Springer US
spellingShingle Haley, Rebecca M.
Gottardi, Riccardo
Langer, Robert S
Mitchell, Michael J.
Cyclodextrins in drug delivery: applications in gene and combination therapy
title Cyclodextrins in drug delivery: applications in gene and combination therapy
title_full Cyclodextrins in drug delivery: applications in gene and combination therapy
title_fullStr Cyclodextrins in drug delivery: applications in gene and combination therapy
title_full_unstemmed Cyclodextrins in drug delivery: applications in gene and combination therapy
title_short Cyclodextrins in drug delivery: applications in gene and combination therapy
title_sort cyclodextrins in drug delivery applications in gene and combination therapy
url https://hdl.handle.net/1721.1/128393
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