科研之友
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摘要
Successful gene therapy depends both on the effective transport and the stable expression of therapeutic genes to produce and regulate disease related proteins. In this context, non-viral gene delivery vehicles are regarded as one of the most promising approaches for the efficient and safe transport of genetic material to and into the target cells. This short review describes the development of novel particulate delivery vehicles based on the biopolymer dextran. This multifunctional platform was designed to safely transport genetic material across cell membranes, followed by an acid triggered release that causes overall high transfection efficiency. The biocompatibility and its unique tunability differentiate this new carrier system from previous particle systems, showing high potential for the treatment of several disease models in RNA interference related applications.
- 出版日期2012
