聚氨基酯作为基因载体的研究进展

王江峰; 鲁莹; 钟延强

聚氨基酯作为基因载体的研究进展

第二军医大学药剂学教研室,上海 200433

Research on poly amino ester as the gene vector

Department of pharmaceutical, Second Military Medical University, Shanghai 200433, China

摘要: 随着基因治疗研究的不断深入,寻求一种高效、安全、靶向表达的载体已成为了基因治疗的核心问题之一。聚氨基酯(poly amino ester,PAE)作为一种新型的阳离子聚合物基因载体,具有原料廉价、合成简单、结构多样、可降解、细胞毒性低和转染效率高等优点,已越来越受到人们的关注。本文将从聚氨基酯的合成途径、理化性质、聚氨基酯/DNA纳米粒的制备及聚氨基酯/DNA纳米粒转染效率和影响因素等方面详细阐述了近年来聚氨基酯作为基因载体的研究进展。
- 聚氨基酯 /
- 非病毒基因载体 /
- 阳离子聚合物
Abstract: With the deepening research of gene therapy, to find an efficient, safe, targeted expression gene vector had become one of the core issues of gene therapy. Poly amino ester (PAE) as a new type of cationic polymer gene carriers, with cheap raw materials, simple synthesis, diversity structure, biodegradability, low toxicity and high efficiency transfection, which had attracted lots of researchers' focus. Recent research of PAE as a gene vector from the poly amino ester synthesis pathway, physical and chemical properties, properties of poly amino ester / DNA nano-particles and transfection efficiency of polyamino ester/DNA nano-particles were summarized in this paper.
- poly amino esters /
- non-viral gene vector /
- cationic polymer

[1]	Sun JY, Anand-Jawa V, Chatterjee S, et al. Immune responses to adeno-associated virus and its recombinant vectors[J]. Gene Ther, 2003,10: 964.
[2]	Gore ME. Adverse effects of gene therapy: gene therapy can cause leukaemia: no shock, mild horror but a probe[J]. Gene Ther, 2003, 10: 4.
[3]	Boussif O, Lezoualc'h F, Zanta, et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine[J]. Proc Natl Acad Sci, 1995, 92: 7297.
[4]	Luo D, Saltzman WM. Synthetic DNA delivery systems[J]. NatBiotechnol, 2000, 18 (1):33.
[5]	Lemkine GF, Demeneix BA. Polyethylenimines for in vivo gene delivery[J]. Curr Opin Mol Ther, 2001, 3: 178.
[6]	Danusso F, Ferruti P. Synthesis of tertiary amine polymers[J]. Polymer, 1970, 11: 88.
[7]	Lynn DM, Robert Langer. Degradable poly(β-amino esters): synthesis, characterization, and self-assembly with plasmid DNA[J]. Am Chem Soc, 2000, 122:10761.
[8]	Lynn DM, Anderson DG, Putnam D, et al. Accelerated discovery of synthetic transfection vectors: parallel synthesis and screening of a degradable polymer library[J]. Am Chem Soc, 2001, 123: 8155.
[9]	Akin Akinc, Anderson DG, Lynn DM, et al. Synthesis of poly(β-amino ester)s optimized for highly effective gene delivery[J]. Bioconjugate Chem, 2003, 14: 979.
[10]	Anderson DG, Lynn DM, and Robert Langer. Semi-automated synthesis and screening of a large library of degradable cationic polymers for gene delivery[J]. Angew Chem Int Ed, 2003, 42: 3153 .
[11]	Zhong Zhiyuan , Song Yan , Johan FJ, et al. A versatile family of degradable non-viral gene carriers based on hyperbranched poly(ester amine)s[J]. Journal of Controlled Release, 2005,109: 317.
[12]	Tae-il Kim, Hyo Jung Seo, Joon Sig Choi, et al. Synthesis of biodegradable cross-linked poly(β-amino ester) for gene delivery and its modification, inducing enhanced transfection efficiency and stepwise degradation[J]. Bioconjugate Chem. 2005, 16:1140.
[13]	Hyun JK, Min SK, Joon SC, et al. Synthesis and characterization of poly (amino ester) for slow biodegradable gene delivery vector[J]. Bioorganic & Medicinal Chemistry, 2007, 15: 1708.
[14]	Liu G, Molas M, Grossmann GA, et al. Biological properties of poly-L-lysine-DNA complexes generated by cooperative binding of the polycation[J]. Biol Chem 2001, 276: 34379.
[15]	Ogris M, Steinlein P, Carotta S, et al. DNA/polyethylenimine transfection particles: influence of ligands, polymer size, and PEGylation on internalization and gene expression[J]. AAPS PharmSci. 2001, 3: 21.
[16]	Anderson DG, Akinc A, Hossain N, et al. Structure/property studies of polymeric gene delivery using a library of poly(beta-amino esters) [J]. Mol Ther, 2005, 11: 426.
[17]	Guo W, Lee RJ. Efficient gene delivery via non-covalent complexes of folic acid and polyethylenimine[J]. Controlled Release 2001, 77: 131.
[18]	Green J, Shi J, Chiu E, et al. Biodegradable polymeric vectors for gene delivery to human endothelial cells[J]. Bioconjugate Chem, 2006, 1: 1162.
[19]	韦卫中.阳离子聚合物纳米基因载体的研究进展[J].医学研究生学报,2004,17(4):335.
[20]	Akinc A, Langer R. Measuring the pH environment of DNA delivered using nonviral vectors: implications for lysosomal trafficking[J]. Biotechnol Bioeng, 2002, 78:503.
[21]	Schaffer DV, Fidelman NA, Dan N, et al. Vector unpacking as a potential barrier for receptor-mediated polyplex gene delivery[J]. Biotechnol Bioeng, 2000, 67: 598.
[22]	Choosakoonkriang S, Lobo BA, Koe GS, et al. Biophysical characterization of PEI/DNA complexes[J]. Pharm Sci, 2003, 92:1710.
[23]	Zugates GT, Nathan CT, Andreas Zumbuehl, et al. Gene delivery properties of end-modified poly(beta-amino ester)s[J]. Bioconjugate Chem. 2007, 18: 1887.
[24]	Zugates G, Anderson D, Little S, et al. Synthesis of poly(beta amino ester)s with thiol-reactive side chains for DNA delivery[J]. Am Chem Soc, 2006, 128: 12726.
[25]	Green JJ, Chiu E, Leshchiner ES, et al. Electrostatic ligand coatings of nanoparticles enable ligand-specific gene delivery to human primary cells[J]. Nano Lett, 2007, 7: 874.

[1]	桂明珠, 李静, 李志玲. 儿童伏立康唑的血药浓度与CYP2C19、CYP2C9和CYP3A5基因多态性的相关性研究 . 药学实践与服务, 2024, 42(): 1-5. doi: 10.12206/j.issn.2097-2024.202402020
[2]	黄韵, 张正银, 金英, 郑怡菁, 李铁军, 孙莉莉. 耐碳青霉烯类肺炎克雷伯菌及大肠埃希菌临床分离株耐药性及耐药基因分析 . 药学实践与服务, 2024, 42(10): 439-444. doi: 10.12206/j.issn.2097-2024.202309059
[3]	刘丽艳, 余小翠, 孙传铎. 纳武利尤单抗治疗非小细胞肺癌有效性及安全性的Meta分析 . 药学实践与服务, 2024, 42(10): 451-456. doi: 10.12206/j.issn.2097-2024.202310044
[4]	杨嘉宁, 赵一颖, 肖伟. 七味脂肝方对非酒精性脂肪性肝炎动物模型的药效学评价 . 药学实践与服务, 2024, 42(9): 389-398. doi: 10.12206/j.issn.2097-2024.202404096
[5]	宋雨桐, 夏德润, 顾珩, 唐少文, 易洪刚, 沃红梅. 帕博利珠单抗与铂类化疗方案在晚期非小细胞肺癌一线治疗中的药物经济学评价 . 药学实践与服务, 2024, 42(8): 334-340. doi: 10.12206/j.issn.2097-2024.202303023
[6]	张艺昕, 关欣怡, 王博宁, 闻俊, 洪战英. 二氢吡啶类钙离子拮抗药物手性分析及其立体选择性药动学研究进展 . 药学实践与服务, 2024, 42(8): 319-324. doi: 10.12206/j.issn.2097-2024.202308062
[7]	王雪莲, 郑斯莉, 李志勇, 罗亨宇, 缪朝玉. 全身过表达人METRNL基因小鼠模型的构建与验证 . 药学实践与服务, 2024, 42(5): 198-202, 222. doi: 10.12206/j.issn.2097-2024.202311014
[8]	冯志惠, 邓仪卿, 叶冰, 安培, 张宏, 张海军. 雀梅藤石油醚提取物诱导三阴性乳腺癌细胞凋亡的实验研究 . 药学实践与服务, 2024, 42(6): 253-259. doi: 10.12206/j.issn.2097-2024.202311055
[9]	王晓飞, 张颖, 顾佳钰, 胡馨儿, 张海, 曹岩. 表面等离子共振传感器的识别元件在医药领域中的研究应用进展 . 药学实践与服务, 2024, 42(): 1-9. doi: 10.12206/j.issn.2097-2024.202309014
[10]	王耀振, 徐灿, 吕顺莉, 田泾, 张东炜. 钾离子竞争性酸阻滞剂的药学特征研究进展 . 药学实践与服务, 2024, 42(7): 278-284. doi: 10.12206/j.issn.2097-2024.202306040
[11]	杨念, 张博乐, 张俊霞, 张振强. 一种中药组合物对ANIT诱导的小鼠胆汁淤积肝损伤的保护作用研究 . 药学实践与服务, 2024, 42(12): 508-511, 519. doi: 10.12206/j.issn.2097-2024.202305008
[12]	姚瑞阳, 于海征, 李耀盺, 张磊. 丹参FBXL 基因家族的鉴定和表达模式分析 . 药学实践与服务, 2024, 42(11): 461-470. doi: 10.12206/j.issn.2097-2024.202407034
[13]	冯婷婷, 张景翔, 王彦, 许维恒, 张俊平. ANXA3基因及蛋白的研究进展 . 药学实践与服务, 2024, 42(): 1-5. doi: 10.12206/j.issn.2097-2024.202309023
[14]	陈炳辰, 佟达丰, 万苗, 闫飞虎, 姚建忠. UPLC-MS/MS法测定小鼠血浆中紫杉醇脂肪酸酯前药及其药代动力学研究 . 药学实践与服务, 2024, 42(8): 341-345. doi: 10.12206/j.issn.2097-2024.202404082
[15]	郭灵怡, 刘艳超, 高路, 刘瑞瑶, 吕权真, 俞媛. 醋酸卡泊芬净单硬脂酸甘油酯纳米粒抗白色念珠菌感染的增效作用研究 . 药学实践与服务, 2024, 42(): 1-8. doi: 10.12206/j.issn.2097-2024.202310043
[16]	陈炳辰, 王思真, 郭贝贝, 杨峰. 紫杉醇棕榈酸酯的合成及其脂质体的制备与处方研究 . 药学实践与服务, 2024, 42(9): 379-384, 410. doi: 10.12206/j.issn.2097-2024.202404062
[17]	崔亚玲, 吴琼, 马良煜, 胡北, 姚东, 许子华. 肝素钠肌醇烟酸酯乳膏中肌醇烟酸酯皮肤药动学研究 . 药学实践与服务, 2024, 42(): 1-5. doi: 10.12206/j.issn.2097-2024.202404006

点击查看大图

计量

文章访问数: 2837
HTML全文浏览量: 268
PDF下载量: 257
被引次数: 0

姓名
邮箱
手机号码
标题
留言内容
验证码

留言板

聚氨基酯作为基因载体的研究进展

Research on poly amino ester as the gene vector

计量

聚氨基酯作为基因载体的研究进展

第二军医大学药剂学教研室,上海 200433

English Abstract

Research on poly amino ester as the gene vector

Department of pharmaceutical, Second Military Medical University, Shanghai 200433, China

全文HTML

目录

留言板

聚氨基酯作为基因载体的研究进展

Research on poly amino ester as the gene vector

计量

出版历程

聚氨基酯作为基因载体的研究进展

第二军医大学药剂学教研室,上海 200433

English Abstract

Research on poly amino ester as the gene vector

Department of pharmaceutical, Second Military Medical University, Shanghai 200433, China

全文HTML

目录