AAV vectors for RNA-based modulation of gene expression

被引:9
作者
Danos, O. [1 ,2 ]
机构
[1] Hop Necker Enfants Malad, Inserm U781, F-75015 Paris, France
[2] Univ Paris 05, F-75015 Paris, France
关键词
AAV vectors; non-coding RNA; antisense; RNA interference; exon-skipping;
D O I
10.1038/gt.2008.69
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
At the post-transcriptional level, gene expression is largely regulated through a network of molecular machines that regulate pre-mRNA maturation integrity, transport, translation and degradation. These processes are based on the formation of nucleoprotein complexes and require the recognition of sequence motifs on the RNA. By masking these targets with complementary RNA sequences forming Watson-Crick base pairing, it is possible to efficiently and specifically impact on the cell phenotype, or to compensate the deleterious effect of mutations. Here we review how the adeno-associated virus technology is being exploited for expressing non-coding RNAs in tissues such as the brain, muscle or liver, in functional genomic studies as well as for the development of novel therapeutic strategies.
引用
收藏
页码:864 / 869
页数:6
相关论文
共 54 条
  • [1] Anti-clarin-1 AAV-delivered ribozyme induced apoptosis in the mouse cochlea
    Aarnisalo, A. A.
    Pietola, L.
    Joensuu, J.
    Isosomppi, J.
    Aarnisalo, P.
    Dinculescu, A.
    Lewin, A. S.
    Flannery, J.
    Hauswirth, W. W.
    Sankila, E.-M.
    Jero, J.
    [J]. HEARING RESEARCH, 2007, 230 (1-2) : 9 - 16
  • [2] AAV-mediated knockdown of phosphotamban teads to improved contractitity and catcium handting in cardiomyocytes
    Andino, Lourdes M.
    Takeda, Morihiko
    Kasahara, Hideko
    Jakymiw, Andrew
    Byrne, Barry J.
    Lewin, Alfred S.
    [J]. JOURNAL OF GENE MEDICINE, 2008, 10 (02) : 132 - 142
  • [3] Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model
    Auricchio, A
    Kobinger, G
    Anand, V
    Hildinger, M
    O'Connor, E
    Maguire, AM
    Wilson, JM
    Bennett, J
    [J]. HUMAN MOLECULAR GENETICS, 2001, 10 (26) : 3075 - 3081
  • [4] In vivo inhibition of hippocampal Ca2+/calmodulin-dependent protein kinase II by RNA interference
    Babcock, AM
    Standing, D
    Bullshields, K
    Schwartz, E
    Paden, CM
    Poulsen, DJ
    [J]. MOLECULAR THERAPY, 2005, 11 (06) : 899 - 905
  • [5] Time-dependent reorganization of brain circuitry underlying long-term memory storage
    Bontempi, B
    Laurent-Demir, C
    Destrade, C
    Jaffard, R
    [J]. NATURE, 1999, 400 (6745) : 671 - 675
  • [6] Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system
    Burger, C
    Gorbatyuk, OS
    Velardo, MJ
    Peden, CS
    Williams, P
    Zolotukhin, S
    Reier, PJ
    Mandel, RJ
    Muzyczka, N
    [J]. MOLECULAR THERAPY, 2004, 10 (02) : 302 - 317
  • [7] VEGF links hippocampal activity with neurogenesis, learning and memory
    Cao, L
    Jiao, XY
    Zuzga, DS
    Liu, YH
    Fong, DM
    Young, D
    During, MJ
    [J]. NATURE GENETICS, 2004, 36 (08) : 827 - 835
  • [8] Lessons from Nature: microRNA-based shRNA libraries
    Chang, Kenneth
    Elledge, Stephen J.
    Hannon, Gregory J.
    [J]. NATURE METHODS, 2006, 3 (09) : 707 - 714
  • [9] CONRAD SE, 1984, CELL, V36, P1007
  • [10] Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model
    Denti, MA
    Rosa, A
    D'Antona, G
    Sthandier, O
    De Angelis, FG
    Nicoletti, C
    Allocca, M
    Pansarasa, O
    Parente, V
    Musarò, A
    Auricchio, A
    Bottinelli, R
    Bozzoni, I
    [J]. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2006, 103 (10) : 3758 - 3763