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
  • [51] RNA interference as a new strategy against viral hepatitis
    Radhakrishnan, SK
    Layden, TJ
    Gartel, AL
    [J]. VIROLOGY, 2004, 323 (02) : 173 - 181
  • [52] Reich S, 2003, MOL VIS, V9, P210
  • [53] A lentivirus-based system to functionally silence genes in primary mammalian cells, stem cells and transgenic mice by RNA interference
    Rubinson, DA
    Dillon, CP
    Kwiatkowski, AV
    Sievers, C
    Yang, LL
    Kopinja, J
    Zhang, MD
    McManus, MT
    Gertler, FB
    Scott, ML
    Van Parijs, L
    [J]. NATURE GENETICS, 2003, 33 (03) : 401 - 406
  • [54] siRNA therapeutics: Big potential from small RNAs
    Ryther, RCC
    Flynt, AS
    Phillips, JA
    Patton, JG
    [J]. GENE THERAPY, 2005, 12 (01) : 5 - 11
  • [55] SHANKAR P, IN PRESS HIV CHEMOTH
  • [56] Rotavirus replication: Plus-sense templates for double-stranded RNA synthesis are made in viroplasms
    Silvestri, LS
    Taraporewala, ZF
    Patton, JT
    [J]. JOURNAL OF VIROLOGY, 2004, 78 (14) : 7763 - 7774
  • [57] Activation of the interferon system by short-interfering RNAs
    Sledz, CA
    Holko, M
    de Veer, MJ
    Silverman, RH
    Williams, BRG
    [J]. NATURE CELL BIOLOGY, 2003, 5 (09) : 834 - 839
  • [58] Sustained small interfering RNA-mediated human immunodeficiency virus type 1 inhibition in primary macrophages
    Song, EW
    Lee, SK
    Dykxhoorn, DM
    Novina, C
    Zhang, D
    Crawford, K
    Cerny, J
    Sharp, PA
    Lieberman, J
    Manjunath, N
    Shankar, P
    [J]. JOURNAL OF VIROLOGY, 2003, 77 (13) : 7174 - 7181
  • [59] RNA interference targeting Fas protects mice from fulminant hepatitis
    Song, EW
    Lee, SK
    Wang, J
    Ince, N
    Ouyang, N
    Min, J
    Chen, JS
    Shankar, P
    Lieberman, J
    [J]. NATURE MEDICINE, 2003, 9 (03) : 347 - 351
  • [60] Crystal structure of argonaute and its implications for RISC slicer activity
    Song, JJ
    Smith, SK
    Hannon, GJ
    Joshua-Tor, L
    [J]. SCIENCE, 2004, 305 (5689) : 1434 - 1437