Highly Branched Poly(β-Amino Esters): Synthesis and Application in Gene Delivery

被引:89
作者
Cutlar, Lara [1 ]
Zhou, Dezhong [1 ]
Gao, Yongsheng [1 ]
Zhao, Tianyu [1 ]
Greiser, Udo [1 ]
Wang, Wei [1 ,2 ]
Wang, Wenxin [1 ,2 ]
机构
[1] Univ Coll Dublin, Charles Inst Dermatol, Sch Med & Med Sci, Dublin 4, Ireland
[2] Tianjin Univ, Sch Mat Sci & Engn, Tianjin 300072, Peoples R China
基金
爱尔兰科学基金会;
关键词
POLYMER LIBRARY APPROACH; IN-VIVO; TRANSGENE EXPRESSION; MOLECULAR-WEIGHT; MINICIRCLE DNA; PLASMID DNA; THERAPY; TRANSFECTION; CHITOSAN; CARRIERS;
D O I
10.1021/acs.biomac.5b00966
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
Highly branched poly(beta-amino esters) (HPAEs) are developed via a facile and controllable "A2+B3/B2" strategy successfully. As nonviral gene delivery vectors, the performance of HPAEs is superior to the well-studied linear counterpart as well as the leading commercial reagent Superfect. When combined with minicircle DNA construct, HPAEs can achieve ultrahigh gene transfection efficiency, especially in keratinocytes.
引用
收藏
页码:2609 / 2617
页数:9
相关论文
共 34 条
[1]   Exploring polyethylenimine-mediated DNA transfection and the proton sponge hypothesis [J].
Akinc, A ;
Thomas, M ;
Klibanov, AM ;
Langer, R .
JOURNAL OF GENE MEDICINE, 2005, 7 (05) :657-663
[2]  
Anderson D. G., 2005, MOL THER, V11, P426
[3]   A polymer library approach to suicide gene therapy for cancer [J].
Anderson, DG ;
Peng, WD ;
Akinc, A ;
Hossain, N ;
Kohn, A ;
Padera, R ;
Langer, R ;
Sawicki, JA .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2004, 101 (45) :16028-16033
[4]   Semi-automated synthesis and screening of a large library of degradable cationic polymers for gene delivery [J].
Anderson, DG ;
Lynn, DM ;
Langer, R .
ANGEWANDTE CHEMIE-INTERNATIONAL EDITION, 2003, 42 (27) :3153-3158
[5]   Efficient Gene Delivery with Osmotically Active and Hyperbranched Poly(ester amine)s [J].
Arote, Rohidas B. ;
Lee, Eun-Sun ;
Jiang, Hu-Lin ;
Kim, You-Kyoung ;
Choi, Yun-Jaie ;
Cho, Myung-Haing ;
Cho, Chong-Su .
BIOCONJUGATE CHEMISTRY, 2009, 20 (12) :2231-2241
[6]   An araC-controlled bacterial cre expression system to produce DNA minicircle vectors for nuclear and mitochondrial gene therapy [J].
Bigger, BW ;
Tolmachov, O ;
Collombet, JM ;
Fragkos, M ;
Palaszewski, I ;
Coutelle, C .
JOURNAL OF BIOLOGICAL CHEMISTRY, 2001, 276 (25) :23018-23027
[7]   Deoxycholic acid-conjugated chitosan oligosaccharide nanoparticles for efficient gene carrier [J].
Chae, SY ;
Son, S ;
Lee, M ;
Jang, MK ;
Nah, JW .
JOURNAL OF CONTROLLED RELEASE, 2005, 109 (1-3) :330-344
[8]   Keratinocyte cell lines derived from severe generalized recessive Epidermolysis Bullosa patients carrying a highly recurrent COL7A1 homozygous mutation: models to assess cell and gene therapies in vitro and in vivo [J].
Chamorro, Cristina ;
Almarza, David ;
Duarte, Blanca ;
Llames, Sara G. ;
Murillas, Rodolfo ;
Garcia, Marta ;
Cigudosa, Juan C. ;
Espinosa-Hevia, Luis ;
Jose Escamez, Maria ;
Mencia, Angeles ;
Meana, Alvaro ;
Garcia-Escudero, Ramon ;
Moro, Rosa ;
Conti, Claudio J. ;
Del Rio, Marcela ;
Larcher, Fernando .
EXPERIMENTAL DERMATOLOGY, 2013, 22 (09) :601-603
[9]   Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo [J].
Chen, ZY ;
He, CY ;
Meuse, L ;
Kay, MA .
GENE THERAPY, 2004, 11 (10) :856-864
[10]   Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo [J].
Chen, ZY ;
He, CY ;
Ehrhardt, A ;
Kay, MA .
MOLECULAR THERAPY, 2003, 8 (03) :495-500