Design and development of polymers for gene delivery

被引:2170
作者
Pack, DW
Hoffman, AS
Pun, S
Stayton, PS
机构
[1] Univ Illinois, Dept Chem & Biomol Engn, Urbana, IL 61801 USA
[2] Univ Washington, Dept Bioengn, Seattle, WA 98195 USA
关键词
D O I
10.1038/nrd1775
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
The lack of safe and efficient gene-delivery methods is a limiting obstacle to human gene therapy. Synthetic gene-delivery agents, although safer than recombinant viruses, generally do not possess the required efficacy. In recent years, a variety of effective polymers have been designed specifically for gene delivery, and much has been learned about their structure-function relationships. With the growing understanding of polymer gene-delivery mechanisms and continued efforts of creative polymer chemists, it is likely that polymer-based gene-delivery systems will become an important tool for human gene therapy.
引用
收藏
页码:581 / 593
页数:13
相关论文
共 187 条
[1]   A powerful nonviral vector for in vivo gene transfer into the adult mammalian brain: Polyethylenimine [J].
Abdallah, B ;
Hassan, A ;
Benoist, C ;
Goula, D ;
Behr, JP ;
Demeneix, BA .
HUMAN GENE THERAPY, 1996, 7 (16) :1947-1954
[2]   Direct real-time molecular scale visualisation of the degradation of condensed DNA complexes exposed to DNase I [J].
Abdelhady, HG ;
Allen, S ;
Davies, MC ;
Roberts, CJ ;
Tendler, SJB ;
Williams, PM .
NUCLEIC ACIDS RESEARCH, 2003, 31 (14) :4001-4005
[3]   Gene therapy for amyotrophic lateral sclerosis and other motor neuron diseases [J].
Alisky, JM ;
Davidson, BL .
HUMAN GENE THERAPY, 2000, 11 (17) :2315-2329
[4]   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
[5]   Enhancement of gene expression by polyamidoamine dendrimer conjugates with α-, β-, and γ-cyclodextrins [J].
Arima, H ;
Kihara, F ;
Hirayama, F ;
Uekama, K .
BIOCONJUGATE CHEMISTRY, 2001, 12 (04) :476-484
[6]   Poly-L-lysine-graft-PEG-comb-type polycation copolymers for gene delivery [J].
Banaszczyk, MG ;
Lollo, CP ;
Kwoh, DY ;
Phillips, AT ;
Amini, A ;
Wu, DP ;
Mullen, PM ;
Coffin, CC ;
Brostoff, SW ;
Carlo, DJ .
JOURNAL OF MACROMOLECULAR SCIENCE-PURE AND APPLIED CHEMISTRY, 1999, A36 (7-8) :1061-1084
[7]  
Behr JP, 1997, CHIMIA, V51, P34
[8]   Transferrin-containing, cyclodextrin polymer-based particles for tumor-targeted gene delivery [J].
Bellocq, NC ;
Pun, SH ;
Jensen, GS ;
Davis, ME .
BIOCONJUGATE CHEMISTRY, 2003, 14 (06) :1122-1132
[9]   Size reduction of galactosylated PEI/DNA complexes improves lectin-mediated gene transfer into hepatocytes [J].
Bettinger, T ;
Remy, JS ;
Erbacher, P .
BIOCONJUGATE CHEMISTRY, 1999, 10 (04) :558-561
[10]   T-LYMPHOCYTE-DIRECTED GENE-THERAPY FOR ADA(-) SCID - INITIAL TRIAL RESULTS AFTER 4 YEARS [J].
BLAESE, RM ;
CULVER, KW ;
MILLER, AD ;
CARTER, CS ;
FLEISHER, T ;
CLERICI, M ;
SHEARER, G ;
CHANG, L ;
CHIANG, YW ;
TOLSTOSHEV, P ;
GREENBLATT, JJ ;
ROSENBERG, SA ;
KLEIN, H ;
BERGER, M ;
MULLEN, CA ;
RAMSEY, WJ ;
MUUL, L ;
MORGAN, RA ;
ANDERSON, WF .
SCIENCE, 1995, 270 (5235) :475-480