Application of Therapeutic Artificial miRNAs in the CNS: Non-Allele-Specific Silencing of Mutant and Wildtype Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice

被引：0
|
作者：
Boudreau, Ryan L. [1 ]
McBride, Jodi L. [1 ]
Martins, Ines [1 ]
Shen, Shihao [1 ]
Xing, Yi [1 ]
Carter, Barrie J. [2 ]
Davidson, Beverly L. [1 ]
机构：
[1] Univ Iowa, Iowa City, IA USA
[2] Targeted Genet, Seattle, WA USA
来源：
MOLECULAR THERAPY | 2009年 / 17卷
关键词：
D O I：
暂无
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
418
引用
收藏
页码：S163 / S164
页数：2
相关论文
共 7 条
  • [1] Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice
    Boudreau, Ryan L.
    McBride, Jodi L.
    Martins, Ines
    Shen, Shihao
    Xing, Yi
    Carter, Barrie J.
    Davidson, Beverly L.
    MOLECULAR THERAPY, 2009, 17 (06) : 1053 - 1063
  • [2] Allele-Specific Silencing of Mutant Huntingtin for Huntington's Disease Therapy
    Mas-Monteys, Alex
    Harper, Scott Q.
    Gilmore, Brian L.
    Staber, Patrick D.
    Schaffer, Chris
    Polisky, Barry
    Vargeese, Chandra
    Davidson, Beverly L.
    MOLECULAR THERAPY, 2006, 13 : S274 - S275
  • [3] Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts
    van Bilsen, P. H. J.
    Jaspers, L.
    Lombardi, M. S.
    Odekerken, J. C. E.
    Burright, E. N.
    Kaemmerer, W. F.
    HUMAN GENE THERAPY, 2008, 19 (07) : 710 - 718
  • [4] Allele-Specific Silencing of Mutant Huntingtin in a Lentiviral-Based Rat Model of Huntington's Disease
    Drouet, Valerie
    Hassig, Raymonde
    Auregan, Gwennaelle
    Dufour, Noelle
    Gorrichon, Kevin
    Brouillet, Emmanuel
    Bonvento, Gilles
    Hantraye, Philippe
    Deglon, Nicole
    MOLECULAR THERAPY, 2009, 17 : S194 - S195
  • [5] Allele-Specific Suppression of Mutant Huntingtin Using Antisense Oligonucleotides: Providing a Therapeutic Option for All Huntington Disease Patients
    Skotte, Niels H.
    Southwell, Amber L.
    Ostergaard, Michael E.
    Carroll, Jeffrey B.
    Warby, Simon C.
    Doty, Crystal N.
    Petoukhov, Eugenia
    Vaid, Kuljeet
    Kordasiewicz, Holly
    Watt, Andrew T.
    Freier, Susan M.
    Hung, Gene
    Seth, Punit P.
    Bennett, C. Frank
    Swayze, Eric E.
    Hayden, Michael R.
    PLOS ONE, 2014, 9 (09):
  • [6] Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models
    Conroy, Faith
    Miller, Rachael
    Alterman, Julia F.
    Hassler, Matthew R.
    Echeverria, Dimas
    Godinho, Bruno M. D. C.
    Knox, Emily G.
    Sapp, Ellen
    Sousa, Jaquelyn
    Yamada, Ken
    Mahmood, Farah
    Boudi, Adel
    Kegel-Gleason, Kimberly
    DiFiglia, Marian
    Aronin, Neil
    Khvorova, Anastasia
    Pfister, Edith L.
    NATURE COMMUNICATIONS, 2022, 13 (01)
  • [7] Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models
    Faith Conroy
    Rachael Miller
    Julia F. Alterman
    Matthew R. Hassler
    Dimas Echeverria
    Bruno M. D. C. Godinho
    Emily G. Knox
    Ellen Sapp
    Jaquelyn Sousa
    Ken Yamada
    Farah Mahmood
    Adel Boudi
    Kimberly Kegel-Gleason
    Marian DiFiglia
    Neil Aronin
    Anastasia Khvorova
    Edith L. Pfister
    Nature Communications, 13
1