Allele-Specific Repression of Mutant Huntingtin Expression By Engineered Zinc Finger Transcriptional Repressors as a Potential Therapy for Huntington's Disease

被引:0
|
作者
Zeitler, Bryan [1 ]
Froelich, Steven [1 ]
Yu, Qi [1 ]
Pearl, Jocelynn [1 ]
Paschon, David E. [1 ]
Miller, Jeffrey C. [1 ]
Li, Davis [1 ]
Marlen, Kimberly [1 ]
Guschin, Dmitry [1 ]
Zhang, Lei [1 ]
Mendel, Matthew [1 ]
Munoz-Sanjuan, Ignacio [2 ]
Rebar, Edward J. [1 ]
Urnov, Fyodor D. [1 ]
Gregory, Philip D. [1 ]
Zhang, H. Steve [1 ]
机构
[1] Sangamo BioSci Inc, Richmond, CA USA
[2] CHDI Fdn Inc, Los Angeles, CA USA
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
603
引用
收藏
页码:S233 / S233
页数:1
相关论文
共 50 条
  • [1] Developing engineered zinc finger transcriptional repressors of Huntingtin as a potential therapy for Huntington's disease
    Zhang, H. S.
    Zeitler, B.
    Pearl, J.
    Yu, Q.
    Cherone, J.
    Paschon, D. E.
    Miller, J. C.
    Zhang, L.
    Mendel, M.
    Laganiere, J.
    Santiago, Y.
    Vu, B. J.
    Urnov, F. D.
    Rebar, E. J.
    Gregory, P. D.
    HUMAN GENE THERAPY, 2012, 23 (10) : A45 - A45
  • [2] Developing Engineered Zinc Finger Repressors of Huntingtin as a Potential Therapy for Huntington's Disease
    Zhang, H. Steve
    Paschon, David E.
    Laganiere, Josee
    Zeitler, Bryan
    Mendel, Matthew
    Cherone, Jennifer
    Yu, Qi
    Pearl, Jocelynn
    Vu, Joe
    Santiago, Yolanda
    Miller, Jeffrey C.
    Rebar, Edward J.
    Zhang, Lei
    Gregory, Philip D.
    MOLECULAR THERAPY, 2012, 20 : S68 - S68
  • [3] 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
  • [4] 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
  • [5] 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
  • [6] Allele-specific silencing of mutant Huntington's disease gene
    Zhang, Yu
    Engelman, Joshua
    Friedlander, Robert M.
    JOURNAL OF NEUROCHEMISTRY, 2009, 108 (01) : 82 - 90
  • [7] Allele-Specific Reduction of the Mutant Huntingtin Allele Using Transcription Activator-Like Effectors in Human Huntington's Disease Fibroblasts
    Fink, Kyle D.
    Deng, Peter
    Gutierrez, Josh
    Anderson, Joseph S.
    Torrest, Audrey
    Komarla, Anvita
    Kalomoiris, Stefanos
    Cary, Whitney
    Anderson, Johnathon D.
    Gruenloh, William
    Duffy, Alexandra
    Tempkin, Teresa
    Annett, Geralyn
    Wheelock, Vicki
    Segal, David J.
    Nolta, Jan A.
    CELL TRANSPLANTATION, 2016, 25 (04) : 677 - 686
  • [8] Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease
    Bryan Zeitler
    Steven Froelich
    Kimberly Marlen
    David A Shivak
    Qi Yu
    Davis Li
    Jocelynn R Pearl
    Jeffrey C Miller
    Lei Zhang
    David E Paschon
    Sarah J Hinkley
    Irina Ankoudinova
    Stephen Lam
    Dmitry Guschin
    Lexi Kopan
    Jennifer M Cherone
    Hoang-Oanh B Nguyen
    Guijuan Qiao
    Yasaman Ataei
    Matthew C Mendel
    Rainier Amora
    Richard Surosky
    Josee Laganiere
    B Joseph Vu
    Anand Narayanan
    Yalda Sedaghat
    Karsten Tillack
    Christina Thiede
    Annette Gärtner
    Seung Kwak
    Jonathan Bard
    Ladislav Mrzljak
    Larry Park
    Taneli Heikkinen
    Kimmo K Lehtimäki
    Marie M Svedberg
    Jenny Häggkvist
    Lenke Tari
    Miklós Tóth
    Andrea Varrone
    Christer Halldin
    Andrea E Kudwa
    Sylvie Ramboz
    Michelle Day
    Jyothisri Kondapalli
    D James Surmeier
    Fyodor D Urnov
    Philip D Gregory
    Edward J Rebar
    Ignacio Muñoz-Sanjuán
    Nature Medicine, 2019, 25 : 1131 - 1142
  • [9] Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington's disease
    Zeitler, Bryan
    Froelich, Steven
    Marlen, Kimberly
    Shivak, David A.
    Yu, Qi
    Li, Davis
    Pearl, Jocelynn R.
    Miller, Jeffrey C.
    Zhang, Lei
    Paschon, David E.
    Hinkley, Sarah J.
    Ankoudinova, Irina
    Lam, Stephen
    Guschin, Dmitry
    Kopan, Lexi
    Cherone, Jennifer M.
    Nguyen, Hoang-Oanh B.
    Qiao, Guijuan
    Ataei, Yasaman
    Mendel, Matthew C.
    Amora, Rainier
    Surosky, Richard
    Laganiere, Josee
    Vu, B. Joseph
    Narayanan, Anand
    Sedaghat, Yalda
    Tillack, Karsten
    Thiede, Christina
    Gaertner, Annette
    Kwak, Seung
    Bard, Jonathan
    Mrzljak, Ladislav
    Park, Larry
    Heikkinen, Taneli
    Lehtimaki, Kimmo K.
    Svedberg, Marie M.
    Haggkvist, Jenny
    Tari, Lenke
    Toth, Miklos
    Varrone, Andrea
    Halldin, Christer
    Kudwa, Andrea E.
    Ramboz, Sylvie
    Day, Michelle
    Kondapalli, Jyothisri
    Surmeier, D. James
    Urnov, Fyodor D.
    Gregory, Philip D.
    Rebar, Edward J.
    Munoz-Sanjuan, Ignacio
    NATURE MEDICINE, 2019, 25 (07) : 1131 - +
  • [10] Neuronal and astrocytic contributions to Huntington's disease dissected with zinc finger protein transcriptional repressors
    Gangwani, Mohitkumar R.
    Soto, Joselyn S.
    Jami-Alahmadi, Yasaman
    Tiwari, Srushti
    Kawaguchi, Riki
    Wohlschlegel, James A.
    Khakh, Baljit S.
    CELL REPORTS, 2023, 42 (01):