Histone-mimetic polyplexes for targeted intracellular delivery during gene transfer

The field of gene therapy has garnered significant interest over the past two decades as a method for revolutionizing the treatment of various diseases such as Alzheimer's, Parkinson's, and many types of cancer. In recent years non-viral methods of delivery have received particular attention because of their low toxicity and improved controllability as compared to viral vectors. However, inefficient trafficking to the nucleus is a common cause of limited DNA delivery. Improving the efficacy of non-viral vectors necessitates thorough understanding of their endocytic uptake and subcellular trafficking behavior. Nature's own mechanisms for gene packaging and transfer have stimulated strong interest in histone proteins. Building on histone biology and recognizing the need for improved control of cellular delivery, we have developed a gene transfer method that utilizes native, histone-based processing pathways.