阳离子聚合物基因治疗研究的发展。
Development of recombinant cationic polymers for gene therapy research.
机构信息
Department of Pharmaceutical Sciences, Center for Integrated Biotechnology, Washington State University, Pullman, 99164, USA.
出版信息
Adv Drug Deliv Rev. 2010 Dec 30;62(15):1524-9. doi: 10.1016/j.addr.2010.04.001. Epub 2010 Apr 14.
Abstract
Cationic polymers created through recombinant DNA technology have the potential to fill a void in the area of gene delivery. The recombinant cationic polymers to be discussed here are amino acid based polymers synthesized in E. coli with the purpose to not only address the major barriers to efficient gene delivery but offer safety, biodegradability, targetability and cost-effectiveness. This review helps the readers to get a better understanding about the evolution of recombinant cationic polymers; and the potential advantages that they could offer over viral and synthetic non-viral vectors for gene delivery. It also discusses some of the major challenges that must be addressed in future studies to turn recombinant polymers into clinically effective gene delivery systems. Recent advances with the biopolymer design suggest that this emerging new class of gene delivery systems has the potential to address some of the major barriers to efficient, safe and cost-effective gene therapy.
摘要
通过重组 DNA 技术创造的阳离子聚合物有可能填补基因传递领域的空白。这里将要讨论的重组阳离子聚合物是在大肠杆菌中合成的基于氨基酸的聚合物，其目的不仅是解决高效基因传递的主要障碍，而且还提供安全性、生物降解性、靶向性和成本效益。本综述有助于读者更好地了解重组阳离子聚合物的发展；以及它们在基因传递方面相对于病毒和合成非病毒载体可能具有的潜在优势。它还讨论了未来研究中必须解决的一些主要挑战，以使重组聚合物转化为临床有效的基因传递系统。生物聚合物设计的最新进展表明，这种新兴的新型基因传递系统有可能解决高效、安全和具有成本效益的基因治疗的一些主要障碍。
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