The prospect of silencing disease using RNA interference

被引:106
作者
Shankar, P
Manjunath, N
Lieberman, J
机构
[1] Harvard Univ, Sch Med, CBR Inst Biomed Res, Boston, MA 02115 USA
[2] Harvard Univ, Sch Med, Dept Pediat, Boston, MA 02115 USA
来源
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION | 2005年 / 293卷 / 11期
关键词
D O I
10.1001/jama.293.11.1367
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
The discovery of RNA interference (RNAi), an endogenous cellular gene-silencing mechanism, has already provided a powerful tool for basic science researchers to study gene function. The subsequent finding that RNAi also operates in mammalian cells has generated excitement regarding potential therapeutic applications. In this article we discuss the basic mechanism of RNA! and the therapeutic opportunities and obstacles for harnessing RNAi for therapy of human disease.
引用
收藏
页码:1367 / 1373
页数:7
相关论文
共 70 条
  • [1] Inhibition of HIV-1 by lentiviral vector-transduced siRNAs in T lymphocytes differentiated in SCID-hu mice and CD34+ progenitor cell-derived macrophages
    Banerjea, A
    Li, MJ
    Bauer, G
    Remling, L
    Lee, NS
    Rossi, J
    Akkina, R
    [J]. MOLECULAR THERAPY, 2003, 8 (01) : 62 - 71
  • [2] MicroRNAs: Genomics, biogenesis, mechanism, and function (Reprinted from Cell, vol 116, pg 281-297, 2004)
    Bartel, David P.
    [J]. CELL, 2007, 131 (04) : 11 - 29
  • [3] Comparison of antisense oligonucleotides and siRNAs in cell culture and in vivo
    Bertrand, JR
    Pottier, M
    Vekris, A
    Opolon, P
    Maksimenko, A
    Malvy, C
    [J]. BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, 2002, 296 (04) : 1000 - 1004
  • [4] Inhibition of respiratory viruses by nasally administered siRNA
    Bitko, V
    Musiyenko, A
    Shulyayeva, O
    Barik, S
    [J]. NATURE MEDICINE, 2005, 11 (01) : 50 - 55
  • [5] Phenotypic silencing of cytoplasmic genes using sequence-specific double-stranded short interfering RNA and its application in the reverse genetics of wild type negative-strand RNA viruses
    Bitko V.
    Barik S.
    [J]. BMC Microbiology, 1 (1) : 1 - 11
  • [6] Stable suppression of tumorigenicity by virus-mediated RNA interference
    Brummelkamp, TR
    Bernards, R
    Agami, R
    [J]. CANCER CELL, 2002, 2 (03) : 243 - 247
  • [7] Loss of the cylindromatosis tumour suppressor inhibits apoptosis by activating NF-κB
    Brummelkamp, TR
    Nijman, SMB
    Dirac, AMG
    Bernards, R
    [J]. NATURE, 2003, 424 (6950) : 797 - 801
  • [8] RNA interference: from model organisms towards therapy for neural and neuromuscular disorders
    Buckingham, SD
    Esmaeili, B
    Wood, M
    Sattelle, DB
    [J]. HUMAN MOLECULAR GENETICS, 2004, 13 : R275 - R288
  • [9] Resistance of human hepatitis delta virus RNAs to dicer activity
    Chang, JH
    Provost, P
    Taylor, JM
    [J]. JOURNAL OF VIROLOGY, 2003, 77 (22) : 11910 - 11917
  • [10] MicroRNAs modulate hematopoietic lineage differentiation
    Chen, CZ
    Li, L
    Lodish, HF
    Bartel, DP
    [J]. SCIENCE, 2004, 303 (5654) : 83 - 86