Kwon E J, Bergen J M, Park I K, Pun S H
Department of Bioengineering, University of Washington, Seattle WA 98195, USA.
J Control Release. 2008 Dec 18;132(3):230-5. doi: 10.1016/j.jconrel.2008.06.012. Epub 2008 Jun 21.
Neuron-targeted nucleic acid delivery systems are important technologies for realizing the potential of gene therapy for nervous system disorders. However, neurons are difficult cells to transfect using non-viral vectors due in part to the specific and unique delivery challenges present in these cells. We have investigated several bioactive peptides for their ability to assist in overcoming delivery barriers in mammalian cells. We summarize here our recent progress in developing and applying peptide-modified polycations for nucleic acid delivery. In addition, we present data demonstrating the potential of using multicomponent, peptide-modified polycations for nucleic acid delivery to neurons.
神经元靶向核酸递送系统是实现神经系统疾病基因治疗潜力的重要技术。然而,由于这些细胞存在特定且独特的递送挑战,使用非病毒载体转染神经元是困难的。我们研究了几种生物活性肽协助克服哺乳动物细胞递送障碍的能力。在此我们总结了在开发和应用肽修饰聚阳离子用于核酸递送方面的最新进展。此外,我们展示了使用多组分肽修饰聚阳离子向神经元递送核酸的潜力的数据。