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Improvement of DNA vector delivery of DOTAP lipoplexes by short-chain aminolipids

Buck, Jonas ; Mueller, Dennis ; Mettal, Ute ; Ackermann, Miriam ; Grisch-Chan, Hiu Man ; Thöny, Beat ; Zumbuehl, Andreas ; Huwyler, Jörg ; Witzigmann, Dominik

In: ACS Omega, 2020, vol. 5, no. 38, p. 24724–24732

Cellular delivery of DNA vectors for the expression of therapeutic proteins is a promising approach to treat monogenic disorders or cancer. Significant efforts in a preclinical and clinical setting have been made to develop potent nonviral gene delivery systems based on lipoplexes composed of permanently cationic lipids. However, transfection efficiency and tolerability of such systems are in...