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

Buck J; Grossen P; Cullis PR; Huwyler J; Witzigmann D

doi:10.1021/acsnano.8b07858

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Journal article

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

Buck J Division of Pharmaceutical Technology, Department of Pharmaceutical Sciences , University of Basel , Klingelbergstrasse 50 , 4056 Basel , Switzerland.
Grossen P Division of Pharmaceutical Technology, Department of Pharmaceutical Sciences , University of Basel , Klingelbergstrasse 50 , 4056 Basel , Switzerland.
Cullis PR Department of Biochemistry and Molecular Biology , University of British Columbia , 2350 Health Sciences Mall , Vancouver , British Columbia V6T 1Z3 , Canada.
Huwyler J Division of Pharmaceutical Technology, Department of Pharmaceutical Sciences , University of Basel , Klingelbergstrasse 50 , 4056 Basel , Switzerland.
Witzigmann D Division of Pharmaceutical Technology, Department of Pharmaceutical Sciences , University of Basel , Klingelbergstrasse 50 , 4056 Basel , Switzerland.

2019-03-26

Published in:

ACS nano. - 2019

English 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 preclinical 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.

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