壳聚糖作为核酸递送载体在基因治疗中的研究进展  

Research progress of chitosan as carriers of nucleic acid in gene therapy

作　　者：李华安 李志扬 林博涵 李汉麒 温鼎声 温露 陈钢[1] LI Hua-an;LI Zhi-yang;LIN Bo-han;LI Han-qi;WEN Ding-sheng;WEN Lu;CHEN Gang(Guangdong Provincial Key Laboratory for Research and Evaluation of Pharmaceutical Preparations,Center for Drug Research and Development,Guangdong Pharmaceutical University,Guangzhou 510006,China;School of Pharmacy,Guangdong Pharmaceutical University,Guangzhou 510006,China)

机构地区：[1]广东药科大学新药研发中心/广东省药物新剂型重点实验室,广州510006 [2]广东药科大学药学院,广州510006

出　　处：《中国新药杂志》2025年第7期713-719,共7页Chinese Journal of New Drugs

基　　金：广东省基础与应用基础研究基金区域联合基金青年基金项目(2022A1515110659)。

摘　　要：壳聚糖作为一种阳离子聚合物,具有良好的生物相容性和较高的阳离子密度,是一种理想的核酸递送载体。壳聚糖结构中含有大量氨基,其核酸转染效率受到脱乙酰度、相对分子质量、pH、血清以及氮磷比等因素的影响。由于出色的递送能力,壳聚糖已经广泛应用于DNA和RNA的递送。此外,壳聚糖也被用于递送CRISPR/Cas9,成为进行基因编辑和疾病治疗的新型手段。尽管如此,壳聚糖及其衍生物作为基因载体在细胞内外仍面临障碍,有待进一步解决。As a type of cationic polymer,chitosan is an ideal carrier for nucleic acid delivery due to its excellent biocompatibility,remarkable biodegradability and high cation density.The structure of chitosan contains a large number of amino groups,and the transfection efficiency of nucleic acid is affected by several factors like deacetylation degree,molecular weight,pH,serum and N/P ratio.Due to its superior delivery abilities,chitosan has been widely used in the delivery of DNA and RNA.In addition,chitosan is also used to deliver CRISPR/Cas9 for gene editing,which is a novel approach to disease treatment.Nevertheless,chitosan and its derivatives as gene carriers still face obstacles at both intracellular and extracellular levels that necessitate further resolution.

关 键 词：壳聚糖 阳离子聚合物 核酸递送 基因治疗 基因编辑 

分 类 号：R943[医药卫生—药剂学]