Polymeric delivery of therapeutic nucleic acids

R Kumar, CF Santa Chalarca, MR Bockman… - Chemical …, 2021 - ACS Publications
The advent of genome editing has transformed the therapeutic landscape for several
debilitating diseases, and the clinical outlook for gene therapeutics has never been more …

The great escape: how cationic polyplexes overcome the endosomal barrier

T Bus, A Traeger, US Schubert - Journal of Materials Chemistry B, 2018 - pubs.rsc.org
The targeted and efficiency-oriented delivery of (therapeutic) nucleic acids raises hope for
successful gene therapy, ie, for the local and individual treatment of acquired and inherited …

The proton sponge hypothesis: Fable or fact?

LMP Vermeulen, SC De Smedt, K Remaut… - European Journal of …, 2018 - Elsevier
In non-viral gene therapy, cationic polymers and lipids are frequently used to encapsulate
macromolecular therapeutics into nanoparticles. During their journey to deliver the cargo to …

The possible “proton sponge” effect of polyethylenimine (PEI) does not include change in lysosomal pH

RV Benjaminsen, MA Mattebjerg, JR Henriksen… - Molecular Therapy, 2013 - cell.com
Polycations such as polyethylenimine (PEI) are used in many novel nonviral vector designs
and there are continuous efforts to increase our mechanistic understanding of their …

Exploring polyethylenimine‐mediated DNA transfection and the proton sponge hypothesis

A Akinc, M Thomas, AM Klibanov… - The Journal of Gene …, 2005 - Wiley Online Library
Background The relatively high transfection efficiency of polyethylenimine (PEI) vectors has
been hypothesized to be due to their ability to avoid trafficking to degradative lysosomes …

Polyethylenimine-based non-viral gene delivery systems

U Lungwitz, M Breunig, T Blunk, A Göpferich - European Journal of …, 2005 - Elsevier
Gene therapy has become a promising strategy for the treatment of many inheritable or
acquired diseases that are currently considered incurable. Non-viral vectors have attracted …

Self‐assembly of hyperbranched polymers and its biomedical applications

Y Zhou, W Huang, J Liu, X Zhu, D Yan - Advanced materials, 2010 - Wiley Online Library
Hyperbranched polymers (HBPs) are highly branched macromolecules with a three‐
dimensional dendritic architecture. Due to their unique topological structure and interesting …

Role of clathrin-and caveolae-mediated endocytosis in gene transfer mediated by lipo-and polyplexes

J Rejman, A Bragonzi, M Conese - Molecular therapy, 2005 - cell.com
We investigated the effects of inhibitors of clathrin-mediated endocytosis (chlorpromazine
and K+ depletion) and of caveolae-mediated uptake (filipin and genistein) on internalization …

Polymer systems for gene delivery—past, present, and future

SY Wong, JM Pelet, D Putnam - Progress in Polymer Science, 2007 - Elsevier
Gene therapy has long sought to treat disease by delivering therapeutic genes to diseased
cells. However, its widespread clinical use has faltered in the face of several challenges …

Molecular hurdles in polyfectin design and mechanistic background to polycation induced cytotoxicity

AC Hunter - Advanced drug delivery reviews, 2006 - Elsevier
Synthetic polymer based Polyfectins (cationic polymer-DNA complex) have received
intensive scientific research as they can potentially circumvent problems associated with …