Optimization of endosome escape activity of multicomponent DNA delivery systems
What is it about?
In this paper we assess the capacity of three fusogenic peptides to promote the endosomal escape of a multicomponent gene delivery system incorporating a cell penetrating peptide [TAT(48-60)] to improve cellular uptake, a cationic poly-L-lysine dendron to condense DNA, and a library of different fusogenic endosome escape peptides [GALA, KALA, HA2(1-20)] to assess their capacity to improve endosome escape and how this affects cell uptake, and toxicity.
Why is it important?
Gene therapy is an exciting means to treat or prevent numerous diseases. Safety issues associated with viral gene delivery vectors have led to interest in non-viral approaches. However, this approach faces numerous barriers to its success, with escape from endosomes after the cellular uptake of such systems representing one of the major barriers to non-viral gene delivery. This paper provides important information about the efficacy and issues associated with the use of various endosome escape peptides.
The following have contributed to this page: Dr Peter Michael Moyle and Yu Wan