Gene therapy holds the promise to provide ultimate cure for many genetic diseases. We developed novel biodegradable star-shaped poly-peptides for gene delivery and discovered that star-shaped polymers showed higher gene transfection efficiency in cell culture than the linear counterparts. What is more intriguing is that the plasmid DNA nanoparticles formed with the star polymers can specifically deliver genes to the lung in mice. It is known that although linear polyethyleneimine (L-PEI) formed plasmid nanoparticles can express reporter genes in the lung, it is mainly due to the particle aggregation, which can cause undesired lung embolism. We demonstrated that our nanoparticles do not aggregate in serum and the lung specificity is due to the fast uptake of these particles into endothelial cells, 90% of which resides in the lung. We are currently evaluating the therapeutic efficacy of these nanoparticles in lung disease models.<br/>

Lung-specific gene delivery using biodegradable polypeptides

251st National Meeting (2016)

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