Overcoming Nonviral Gene Delivery Barriers: Perspective and Future

被引:329
作者
Jones, Charles H. [1 ]
Chen, Chih-Kuang [1 ]
Ravikrishnan, Anitha [1 ]
Rane, Snehal [1 ]
Pfeifer, Blaine A. [1 ]
机构
[1] SUNY Buffalo, Dept Chem & Biol Engn, Buffalo, NY 14260 USA
关键词
gene delivery; gene therapy; nonviral vectors; lipoplexes; polyplexes; NUCLEIC-ACID DELIVERY; RING-OPENING POLYMERIZATION; MINICIRCLE DNA VECTORS; PROTEIN-KINASE-C; CATIONIC LIPIDS; IN-VIVO; SIRNA DELIVERY; PLASMID DNA; TRANSFECTION EFFICIENCY; MOLECULAR-WEIGHT;
D O I
10.1021/mp400467x
中图分类号
R-3 [医学研究方法]; R3 [基础医学];
学科分类号
1001 ;
摘要
A key end goal of gene delivery research is to develop clinically relevant vectors that can be used to combat elusive diseases such as AIDS. Despite promising engineering strategies, efficiency and ultimately gene modulation efficacy of nonviral vectors have been hindered by numerous in vitro and in vivo barriers that have resulted in subviral performance. In this perspective, we concentrate on the gene delivery barriers associated with the two most common classes of nonviral vectors, cationic-based lipids and polymers. We present the existing delivery barriers and summarize current vector-specific strategies to overcome said barriers.
引用
收藏
页码:4082 / 4098
页数:17
相关论文
共 141 条
[1]   SELECTION OF DNA CLONES WITH ENHANCER SEQUENCES [J].
ASOH, S ;
LEEKWON, W ;
MOURADIAN, MM ;
NIRENBERG, M .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1994, 91 (15) :6982-6986
[2]   Effect of Thiol Pendant Conjugates on Plasmid DNA Binding, Release, and Stability of Polymeric Delivery Vectors [J].
Bacalocostantis, Irene ;
Mane, Viraj P. ;
Kang, Michael S. ;
Goodley, Addison S. ;
Muro, Silvia ;
Kofinas, Peter .
BIOMACROMOLECULES, 2012, 13 (05) :1331-1339
[3]   Delivery of bacterial artificial chromosomes into mammalian cells with psoralen-inactivated adenovirus carrier [J].
Baker, A ;
Cotten, M .
NUCLEIC ACIDS RESEARCH, 1997, 25 (10) :1950-1956
[4]   Design, synthesis, and transfection biology of novel cationic glycolipids for use in liposomal gene delivery [J].
Banerjee, R ;
Mahidhar, YV ;
Chaudhuri, A ;
Gopal, V ;
Rao, NM .
JOURNAL OF MEDICINAL CHEMISTRY, 2001, 44 (24) :4176-4185
[5]   The Possible "Proton Sponge" Effect of Polyethylenimine (PEI) Does Not Include Change in Lysosomal pH [J].
Benjaminsen, Rikke V. ;
Mattebjerg, Maria A. ;
Henriksen, Jonas R. ;
Moghimi, S. Moein ;
Andresen, Thomas L. .
MOLECULAR THERAPY, 2013, 21 (01) :149-157
[6]   Cationic lipid-mediated gene delivery to murine lung: Correlation of lipid hydration with in vivo transfection activity [J].
Bennett, MJ ;
Aberle, AM ;
Balasubramaniam, RP ;
Malone, JG ;
Malone, RW ;
Nantz, MH .
JOURNAL OF MEDICINAL CHEMISTRY, 1997, 40 (25) :4069-4078
[7]   Advances in gene delivery through molecular design of cationic lipids [J].
Bhattacharya, Santanu ;
Bajaj, Avinash .
CHEMICAL COMMUNICATIONS, 2009, (31) :4632-4656
[8]  
Bloomfield VA, 1997, BIOPOLYMERS, V44, P269, DOI 10.1002/(SICI)1097-0282(1997)44:3<269::AID-BIP6>3.0.CO
[9]  
2-T
[10]   INHIBITION OF PROTEIN-KINASE-C BY CATIONIC AMPHIPHILES [J].
BOTTEGA, R ;
EPAND, RM .
BIOCHEMISTRY, 1992, 31 (37) :9025-9030