Therapeutic siRNAs

被引：98
作者：
Sioud, M [1 ]
机构：
[1] Norwegian Radium Hosp, Dept Immunol, Mol Med Grp, N-310 Oslo, Norway
来源：
TRENDS IN PHARMACOLOGICAL SCIENCES | 2004年 / 25卷 / 01期
关键词：
D O I：
10.1016/j.tips.2003.11.006
中图分类号：
R9 [药学];
学科分类号：
1007 ;
摘要：
The ability of small-interfering RNAs (siRNAs) to silence gene expression in somatic mammalian cells has provided researchers with a novel tool to block the expression of disease-causing genes, provided that their mRNA sequences are known. siRNAs can be delivered to cells either exogenously as synthetic agents or endogenously as gene-encoding siRNAs. Recent studies demonstrate the general application of siRNAs to silence gene expression in a range of cell types and in whole mammals. Beyond their value for dissecting gene functions and target validation, siRNAs also hold great potential as gene-specific therapeutic agents.
引用
收藏
页码：22 / 28
页数：7
相关论文
共 65 条
  • [1] Stable suppression of tumorigenicity by virus-mediated RNA interference
    Brummelkamp, TR
    Bernards, R
    Agami, R
    [J]. CANCER CELL, 2002, 2 (03) : 243 - 247
  • [2] A system for stable expression of short interfering RNAs in mammalian cells
    Brummelkamp, TR
    Bernards, R
    Agami, R
    [J]. SCIENCE, 2002, 296 (5567) : 550 - 553
  • [3] Rescuing the function of mutant p53
    Bullock, AN
    Fersht, A
    [J]. NATURE REVIEWS CANCER, 2001, 1 (01) : 68 - 76
  • [4] Specific inhibition of gene expression by small double-stranded RNAs in invertebrate and vertebrate systems
    Caplen, NJ
    Parrish, S
    Imani, F
    Fire, A
    Morgan, RA
    [J]. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2001, 98 (17) : 9742 - 9747
  • [5] Inhibition of HIV-1 infection by small interfering RNA-mediated RNA interference
    Capodici, J
    Karikó, K
    Weissman, D
    [J]. JOURNAL OF IMMUNOLOGY, 2002, 169 (09) : 5196 - 5201
  • [6] RNA interference is a functional pathway with therapeutic potential in human myeloid leukemia cell lines
    Cioca, DP
    Aoki, Y
    Kiyosawa, K
    [J]. CANCER GENE THERAPY, 2003, 10 (02) : 125 - 133
  • [7] Potent and specific inhibition of human immunodeficiency virus type 1 replication by RNA interference
    Coburn, GA
    Cullen, BR
    [J]. JOURNAL OF VIROLOGY, 2002, 76 (18) : 9225 - 9231
  • [8] The molecular biology of chronic myeloid leukemia
    Deininger, MWN
    Goldman, JM
    Melo, JV
    [J]. BLOOD, 2000, 96 (10) : 3343 - 3356
  • [9] Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
    Elbashir, SM
    Harborth, J
    Lendeckel, W
    Yalcin, A
    Weber, K
    Tuschl, T
    [J]. NATURE, 2001, 411 (6836) : 494 - 498
  • [10] Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
    Fire, A
    Xu, SQ
    Montgomery, MK
    Kostas, SA
    Driver, SE
    Mello, CC
    [J]. NATURE, 1998, 391 (6669) : 806 - 811
1234567