Cai Xue, Conley Shannon, Naash Muna
Department of Cell Biology, University of Oklahoma Health Sciences Center, 940 Stanton L. Young Boulevard, BMSB 781, Oklahoma City, OK 73104, USA.
Vision Res. 2008 Feb;48(3):319-24. doi: 10.1016/j.visres.2007.07.012. Epub 2007 Sep 6.
The use of nanoparticles as carriers for the delivery of therapeutic materials to target tissues has became popular in recent years and has demonstrated great potentials for the treatments of a wide range of diseases. In this review, we summarize the advantages of nanotechnology as a common gene delivery strategy with emphasis on ocular therapy. Particular attention is paid to the CK30-PEG compacted DNA nanoparticles that have been successfully tested in the eye, lung, and brain. These particles resulted in higher transfection efficiency and longer duration of expression than other non-viral vectors without any toxicity or other side effects. They have been safely used clinically and are efficient for a broad range of gene therapy applications. The review also discusses mechanisms of nanoparticle uptake and internalization by cells, obstacles and limitations to the use of this technology, as well as novel methodologies to optimize nanoparticle driven gene expression.
近年来，使用纳米颗粒作为将治疗材料递送至靶组织的载体已变得流行起来，并已在多种疾病的治疗中显示出巨大潜力。在本综述中，我们总结了纳米技术作为一种常见基因递送策略的优势，重点是眼部治疗。特别关注已在眼睛、肺部和大脑中成功测试的CK30-PEG压缩DNA纳米颗粒。与其他非病毒载体相比，这些颗粒具有更高的转染效率和更长的表达持续时间，且无任何毒性或其他副作用。它们已在临床上安全使用，并且对广泛的基因治疗应用有效。该综述还讨论了细胞摄取和内化纳米颗粒的机制、使用该技术的障碍和局限性，以及优化纳米颗粒驱动基因表达的新方法。
