Goodwin Tyler, Huang Leaf
Division of Molecular Pharmaceutics and Center for Nanotechnology in Drug Delivery, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Adv Genet. 2014;88:1-12. doi: 10.1016/B978-0-12-800148-6.00001-8.
Gene therapy, once thought to be the future of medicine, has reached the beginning stages of exponential growth. Many types of diseases are now being studied and treated in clinical trials through various gene delivery vectors. It appears that the future is here, and gene therapy is just beginning to revolutionize the way patients are treated. However, as promising as these ongoing treatments and clinical trials are, there are many more barriers and challenges that need to be addressed and understood in order to continue this positive growth. Our knowledge of these challenging factors such as gene uptake and expression should be expanded in order to improve existing delivery systems. This chapter will provide a brief overview on recent advances in the field of nonviral vectors for gene therapy as well as point out some novel vectors that have assisted in the extraordinary growth of nonviral gene therapy as we know it today.
基因治疗曾被认为是医学的未来，如今已进入指数增长的初始阶段。目前，许多类型的疾病正在通过各种基因递送载体在临床试验中进行研究和治疗。未来似乎已然到来，基因治疗刚刚开始彻底改变治疗患者的方式。然而，尽管这些正在进行的治疗和临床试验前景广阔，但为了延续这种积极的发展态势，仍有许多障碍和挑战需要加以应对和理解。我们对诸如基因摄取和表达等具有挑战性因素的认识应予以扩展，以改进现有的递送系统。本章将简要概述基因治疗中非病毒载体领域的最新进展，并指出一些推动了我们如今所知的非病毒基因治疗显著发展的新型载体。
