Gene therapy is a promising approach for disease prevention and therapy. Efficient gene delivery is an important
factor limiting gene delivery. Due to the poor stability of nucleotide molecules in vivo, they require an association
with delivery systems to overcome extracellular and intracellular barriers and allow access to the site of action. In this review,
we discuss the challenges currently encountered in the delivery of DNA and RNAi-based therapies using the most
common cationic polymer assemblies including polylysines, chitosan, polyethylenimines, dendrimers, cyclodextrins and
illustrate with examples how chemical modification of cationic assemblies may contribute to their successful application
in the clinic.
Keywords: Cationic assemblies, cationic polymers, complexes, DNA delivery, gene delivery, RNA interference, siRNA delivery.
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