Cas9 targeting of toxic foci of RNA repeats

被引：0
|
作者：
Denis Furling
机构：
[1] Sorbonne Université,
[2] Inserm,undefined
[3] Institut de Myologie,undefined
[4] Centre de Recherche en Myologie,undefined
来源：
Nature Biomedical Engineering | 2021年 / 5卷
关键词：
D O I：
暂无
中图分类号：
学科分类号：
摘要：
The sustained expression of RNA-targeting Cas9 delivered intramuscularly or systemically by adeno-associated viral vectors eliminates pathogenic foci of expanded-repeat transcripts and reverses muscle-disease phenotypes in mouse models of myotonic dystrophy type 1.
引用
收藏
页码：130 / 131
页数：1
相关论文
共 50 条
  • [1] Cas9 targeting of toxic foci of RNA repeats
    Furling, Denis
    NATURE BIOMEDICAL ENGINEERING, 2021, 5 (02) : 130 - 131
  • [2] RNA targeting by Cas9
    Eytan Zlotorynski
    Nature Reviews Molecular Cell Biology, 2014, 15 (11) : 701 - 701
  • [3] Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9
    Batra, Ranjan
    Nelles, David A.
    Pirie, Elaine
    Blue, Steven M.
    Marina, Ryan J.
    Wang, Harrison
    Chaim, Isaac A.
    Thomas, James D.
    Zhang, Nigel
    Nguyen, Vu
    Aigner, Stefan
    Markmiller, Sebastian
    Xia, Guangbin
    Corbett, Kevin D.
    Swanson, Maurice S.
    Yeo, Gene W.
    CELL, 2017, 170 (05) : 899 - +
  • [4] Targeting Cas9
    Rusk, Nicole
    NATURE METHODS, 2018, 15 (07) : 481 - 481
  • [5] Targeting Cas9
    Nicole Rusk
    Nature Methods, 2018, 15 : 481 - 481
  • [6] Cleaving RNA with Cas9
    Ghodsizadeh, Odelia
    NATURE METHODS, 2014, 11 (11) : 1090 - 1090
  • [7] Cleaving RNA with Cas9
    Odelia Ghodsizadeh
    Nature Methods, 2014, 11 : 1090 - 1090
  • [8] DNA targeting specificity of RNA-guided Cas9 nucleases
    Patrick D Hsu
    David A Scott
    Joshua A Weinstein
    F Ann Ran
    Silvana Konermann
    Vineeta Agarwala
    Yinqing Li
    Eli J Fine
    Xuebing Wu
    Ophir Shalem
    Thomas J Cradick
    Luciano A Marraffini
    Gang Bao
    Feng Zhang
    Nature Biotechnology, 2013, 31 : 827 - 832
  • [9] DNA targeting specificity of RNA-guided Cas9 nucleases
    Hsu, Patrick D.
    Scott, David A.
    Weinstein, Joshua A.
    Ran, F. Ann
    Konermann, Silvana
    Agarwala, Vineeta
    Li, Yinqing
    Fine, Eli J.
    Wu, Xuebing
    Shalem, Ophir
    Cradick, Thomas J.
    Marraffini, Luciano A.
    Bao, Gang
    Zhang, Feng
    NATURE BIOTECHNOLOGY, 2013, 31 (09) : 827 - +
  • [10] Cas9 Targeting and the CRISPR Revolution
    Barrangou, Rodolphe
    SCIENCE, 2014, 344 (6185) : 707 - 708
12345