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
  • [1] Author Correction: Targeted exon skipping with AAV-mediated split adenine base editors
    Jackson Winter
    Alan Luu
    Michael Gapinske
    Sony Manandhar
    Shraddha Shirguppe
    Wendy S. Woods
    Jun S. Song
    Pablo Perez-Pinera
    Cell Discovery, 5
  • [2] Targeted exon skipping with AAV-mediated split adenine base editors
    Winter, Jackson
    Luu, Alan
    Gapinske, Michael
    Manandhar, Sony
    Shirguppe, Shraddha
    Woods, Wendy S.
    Song, Jun S.
    Perez-Pinera, Pablo
    CELL DISCOVERY, 2019, 5 (1)
  • [3] Targeted exon skipping with AAV-mediated split adenine base editors
    Jackson Winter
    Alan Luu
    Michael Gapinske
    Sony Manandhar
    Shraddha Shirguppe
    Wendy S. Woods
    Jun S. Song
    Pablo Perez-Pinera
    Cell Discovery, 5
  • [4] Targeted exon skipping with AAV-mediated split adenine base editors (vol 5, 41, 2019)
    Winter, Jackson
    Luu, Alan
    Gapinske, Michael
    Manandhar, Sony
    Shirguppe, Shraddha
    Woods, Wendy S.
    Song, Jun S.
    Perez-Pinera, Pablo
    CELL DISCOVERY, 2019, 5 (1)
  • [5] A Universal Correction Strategy for α-Thalassemia Using CRISPR/AAV-Mediated Genome Editing
    Cromer, M. Kyle
    Wienert, Beeke
    Hampton, Jessica P.
    Majeti, Kiran R.
    Conklin, Bruce R.
    Porteus, Matthew H.
    MacKenzie, Tippi C.
    MOLECULAR THERAPY, 2022, 30 (04) : 328 - 328
  • [6] Correcting Duchenne Muscular Dystrophy Using AAV-Mediated Split Base Editors
    Gapinske, Michael
    Winter, Jackson
    Kostan, Kurt A.
    Busza, Anna
    Woods, Wendy S.
    Perez-Pinera, Pablo
    MOLECULAR THERAPY, 2020, 28 (04) : 279 - 279
  • [7] AAV-mediated in vivo genome editing in vascular endothelial cells
    Wu, Wenyi
    Yang, Yanhui
    Yao, Fei
    Dong, Lijun
    Xia, Xiaobo
    Zhang, Shaochong
    Lei, Hetian
    METHODS, 2021, 194 : 12 - 17
  • [8] AAV-mediated somatic genetic correction of epidermolysis bullosa
    Melo, S. P.
    Lisowski, L.
    Bashkirova, L.
    Kay, M. A.
    Oro, A. E.
    JOURNAL OF INVESTIGATIVE DERMATOLOGY, 2013, 133 : S139 - S139
  • [9] A split and inducible adenine base editor for precise in vivo base editing
    Zeng, Hongzhi
    Yuan, Qichen
    Peng, Fei
    Ma, Dacheng
    Lingineni, Ananya
    Chee, Kelly
    Gilberd, Peretz
    Osikpa, Emmanuel C.
    Sun, Zheng
    Gao, Xue
    NATURE COMMUNICATIONS, 2023, 14 (01)
  • [10] A split and inducible adenine base editor for precise in vivo base editing
    Hongzhi Zeng
    Qichen Yuan
    Fei Peng
    Dacheng Ma
    Ananya Lingineni
    Kelly Chee
    Peretz Gilberd
    Emmanuel C. Osikpa
    Zheng Sun
    Xue Gao
    Nature Communications, 14 (1)
12345