New Dendritic Beta-Cyclodextrin/Triazole System for DNA Delivery

摘要

The safe and efficient delivery of therapeutic DNA into cells is a fundamental obstacle to the clinical success of gene therapy. The danger associated with viral vectors has invoked the need for the design of non-viral vectors. Polymers and dendrimers are currently being investigated for delivery of DNA. These synthetic materials offer many advantages over viruses since they can be easily synthesized on a larger scale and are cost effective. Polycations such as polyethylenimine (PEI) and poly(aminoamine) (PAMAM) dendrimer are synthetic non-viral vectors that effectively bind DNA through electrostatic interactions and compact DNA into polyplexes (complexes formed by DNA and the vector).1 These complexes can be taken up by cells through the endocytotic pathway. Even though these materials have high delivery efficiency, they are found to be cytotoxic.2 Several studies have shown that the polymer structure plays a vital role in gene delivery efficiency and cytotoxicity.