In Vivo Genetic Eye Disease Correction Using Split AAV-Mediated Adenine Base Editing

被引：0
作者：
Sullivan, Jack [1 ]
Muller, Alissa [2 ,3 ]
Schwarzer, Wibke [2 ,3 ]
Wang, Mantian [2 ,3 ]
Park-Windhol, Cindy [1 ]
Hasler, Pascal [3 ]
Moreau, Pierre-Henri [4 ]
Azoulay, Thierry [5 ]
Janeschitz-Kriegl, Lucas [2 ,3 ]
Szabo, Arnold [6 ]
Scholl, Hendrik P. N. [2 ,3 ]
Roska, Botond [2 ,3 ]
Gyorgy, Bence [2 ,3 ]
Bryson, David I. [1 ]
Ciaramella, Giuseppe [1 ]
机构：
[1] Beam Therapeut, Cambridge, MA USA
[2] Inst Mol & Clin Ophthalmol Basel, Basel, Switzerland
[3] Univ Basel, Dept Ophthalmol, Basel, Switzerland
[4] Univ Strasbourg, SILABE, Niederhausbergen, France
[5] Clin Vet Agoravet, Strasbourg, France
[6] Semmelweis Univ, Dept Anat, Budapest, Hungary
来源：
MOLECULAR THERAPY | 2023年 / 31卷 / 04期
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：
308
引用
收藏
页码：168 / 168
页数：1
相关论文
共 50 条
  • [31] AAV-mediated knockdown of Peripherin-2 in vivo using miRNA-based hairpins
    Georgiadis, A.
    Tschernutter, M.
    Bainbridge, J. W. B.
    Robbie, S. J.
    McIntosh, J.
    Nathwani, A. C.
    Smith, A. J.
    Ali, R. R.
    GENE THERAPY, 2010, 17 (04) : 486 - 493
  • [32] AAV-mediated knockdown of Peripherin-2 in vivo using miRNA-based hairpins
    A Georgiadis
    M Tschernutter
    J W B Bainbridge
    S J Robbie
    J McIntosh
    A C Nathwani
    A J Smith
    R R Ali
    Gene Therapy, 2010, 17 : 486 - 493
  • [33] AAV-mediated in vivo knockdown of luciferase using combinatorial RNAi and U1i
    Koornneef, A.
    van Logtenstein, R.
    Timmermans, E.
    Pisas, L.
    Blits, B.
    Abad, X.
    Fortes, P.
    Petry, H.
    Konstantinova, P.
    Ritsema, T.
    GENE THERAPY, 2011, 18 (09) : 929 - 935
  • [34] AAV-Mediated Gene Supplementation Provides Thorough Correction in Two Models of Vanishing White Matter Disease
    Herstine, Jessica A.
    Stevenson, Tamara J.
    Chornyy, Sergiy
    Chang, Pi-Kai
    Rediger, Jessica
    Wentz, Julia
    Pyne, Nettie
    Sunshine, Alex
    Scholl, Erika
    Vetter, Tatyana A.
    Flanigan, Kevin M.
    Bonkowsky, Joshua
    Bradbury, Allison M.
    MOLECULAR THERAPY, 2024, 32 (04) : 317 - 317
  • [35] Publisher Correction: Precision genome editing using cytosine and adenine base editors in mammalian cells
    Tony P. Huang
    Gregory A. Newby
    David R. Liu
    Nature Protocols, 2021, 16 : 5740 - 5740
  • [36] Long-term correction of canine FVII deficiency using AAV-mediated expression of zymogen FVII
    Marcos-Contreras, O. A.
    Smith, S. M.
    Bellinger, D. A.
    Merricks, E.
    Faella, A.
    Pavani, G.
    Zhou, S.
    Nichols, T. C.
    High, K. A.
    Margaritis, P.
    JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 2015, 13 : 162 - 162
  • [37] AAV-mediated in vivo knock-down of luciferase using RNAi and U1i
    Koornneef, A.
    van Logtenstein, R.
    Timmermans, E.
    Pisas, L.
    Blits, B.
    Abad, X.
    Fortes, P.
    Petry, H.
    Konstantinova, P.
    Ritsema, T.
    HUMAN GENE THERAPY, 2010, 21 (10) : 1443 - 1444
  • [38] AAV-Mediated PPT1 Replacement and Cross Correction for Treating CLN1 Batten Disease
    Alam, Suhail
    Khatiwada, Apeksha
    Cohen, Daniel M.
    Peterson, Drew R.
    White, John
    Derby, Melissa
    Wang, Bo
    Nayal, Mohamad
    Becker, Mallory
    Benakanakere, Manju
    Beck, Heena
    Li, Charlie
    Gentzel, Renee
    McBride, Jodi L.
    Biferi, Maria Grazia
    Ramsburg, Elizabeth
    MOLECULAR THERAPY, 2024, 32 (04) : 735 - 735
  • [39] In vivo correction of a metabolic liver disease by AAV8-mediated homologous recombination
    Wursthorn, Karsten
    Storm, Terry
    Kay, Mark
    Feingold, Milton
    Grompe, Markus
    HEPATOLOGY, 2006, 44 (04) : 433A - 433A
  • [40] In Vivo Correction of a Metabolic Liver Disease by AAV8-Mediated Homologous Recombination
    Wursthorn, Karsten
    Storm, Terry
    Kay, Mark A.
    Finegold, Milton
    Grompe, Markus
    MOLECULAR THERAPY, 2006, 13 : S311 - S312
12345