Lipid-Based DNA Therapeutics - Hallmarks of Non-Viral Gene Delivery

Buck, Jonas and Grossen, Philip and Cullis, Pieter R. and Huwyler, Jörg and Witzigmann, Dominik. (2019) Lipid-Based DNA Therapeutics - Hallmarks of Non-Viral Gene Delivery. ACS Nano, 13 (4). pp. 3754-3782.

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Official URL: https://edoc.unibas.ch/70350/

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Gene therapy is a promising strategy for the treatment of monogenic disorders. Non-viral gene delivery systems including lipid-based DNA therapeutics offer the opportunity to deliver an encoding gene sequence specifically to the target tissue and thus enable the expression of therapeutic proteins in diseased cells. Currently, available gene delivery approaches based on DNA are inefficient and require improvements to achieve clinical utility. In this review, we discuss state-of-the-art lipid-based DNA delivery systems that have been investigated in a pre-clinical setting. We emphasize factors influencing the delivery and subsequent gene expression in vitro, ex vivo, and in vivo. In addition, we cover aspects of nanoparticle engineering and optimization for DNA therapeutics. Finally, we highlight achievements of lipid-based DNA therapies in clinical trials.
Faculties and Departments:05 Faculty of Science > Departement Pharmazeutische Wissenschaften > Pharmazie > Pharmaceutical Technology (Huwyler)
UniBasel Contributors:Huwyler, Jörg and Buck, Jonas and Grossen, Philip and Witzigmann, Dominik
Item Type:Article, refereed
Article Subtype:Research Article
Publisher:American Chemical Society
Note:Publication type according to Uni Basel Research Database: Journal article
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Last Modified:17 Aug 2020 11:44
Deposited On:17 Aug 2020 11:44

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