School of Materials Science & Engineering, Nanyang Technological University, Singapore 639798, Singapore.
Mar Drugs. 2019 Jun 25;17(6):381. doi: 10.3390/md17060381.
Approximately 4000 diseases are associated with malfunctioning genes in a particular cell type. Gene-based therapy provides a platform to modify the disease-causing genes expression at the cellular level to treat pathological conditions. However, gene delivery is challenging as these therapeutic genes need to overcome several physiological and intracellular barriers in order, to reach the target cells. Over the years, efforts have been dedicated to develop efficient gene delivery vectors to overcome these systemic barriers. Chitosan, a versatile polysaccharide, is an attractive non-viral vector material for gene delivery mainly due to its cationic nature, biodegradability and biocompatibility. The present review discusses the design factors that are critical for efficient gene delivery/transfection and highlights the recent progress of gene therapy using chitosan-based carriers.
大约有 4000 种疾病与特定细胞类型中基因的功能障碍有关。基于基因的治疗为在细胞水平上修饰致病基因的表达提供了一个平台,以治疗病理状况。然而,基因传递是具有挑战性的,因为这些治疗基因需要克服几个生理和细胞内的障碍,以到达靶细胞。多年来,人们一直致力于开发有效的基因传递载体,以克服这些系统障碍。壳聚糖是一种多功能多糖,由于其阳离子性质、可生物降解性和生物相容性,是一种有吸引力的非病毒载体材料,可用于基因传递。本综述讨论了高效基因传递/转染的关键设计因素,并强调了基于壳聚糖的载体在基因治疗中的最新进展。