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
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
  • [41] Publisher Correction: Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing
    Susie Suh
    Elliot H. Choi
    Henri Leinonen
    Andrzej T. Foik
    Gregory A. Newby
    Wei-Hsi Yeh
    Zhiqian Dong
    Philip D. Kiser
    David C. Lyon
    David R. Liu
    Krzysztof Palczewski
    Nature Biomedical Engineering, 2020, 4 : 1119 - 1119
  • [42] Production of MSTN knockout porcine cells using adenine base-editing-mediated exon skipping
    Yang, Shuai-peng
    Zhu, Xiang-xing
    Qu, Zi-xiao
    Chen, Cai-yue
    Wu, Yao-bing
    Wu, Yue
    Luo, Zi-dan
    Wang, Xin-yi
    He, Chu-yu
    Fang, Jia-wen
    Wang, Ling-qi
    Hong, Guang-long
    Zheng, Shu-tao
    Zeng, Jie-mei
    Yan, Ai-fen
    Feng, Juan
    Liu, Lian
    Zhang, Xiao-li
    Zhang, Li-gang
    Miao, Kai
    Tang, Dong-sheng
    IN VITRO CELLULAR & DEVELOPMENTAL BIOLOGY-ANIMAL, 2023, 59 (04) : 241 - 255
  • [43] Production of MSTN knockout porcine cells using adenine base-editing-mediated exon skipping
    Shuai-peng Yang
    Xiang-xing Zhu
    Zi-xiao Qu
    Cai-yue Chen
    Yao-bing Wu
    Yue Wu
    Zi-dan Luo
    Xin-yi Wang
    Chu-yu He
    Jia-wen Fang
    Ling-qi Wang
    Guang-long Hong
    Shu-tao Zheng
    Jie-mei Zeng
    Ai-fen Yan
    Juan Feng
    Lian Liu
    Xiao-li Zhang
    Li-gang Zhang
    Kai Miao
    Dong-sheng Tang
    In Vitro Cellular & Developmental Biology - Animal, 2023, 59 : 241 - 255
  • [44] Utilizing AAV-mediated LEAPER 2.0 for programmable RNA editing in non-human primates and nonsense mutation correction in humanized Hurler syndrome mice
    Zongyi Yi
    Yanxia Zhao
    Zexuan Yi
    Yongjian Zhang
    Gangbin Tang
    Xiaoxue Zhang
    Huixian Tang
    Wei Zhang
    Ying Zhao
    Huayuan Xu
    Yuyang Nie
    Xueqing Sun
    Lijun Xing
    Lian Dai
    Pengfei Yuan
    Wensheng Wei
    Genome Biology, 24
  • [45] Utilizing AAV-mediated LEAPER 2.0 for programmable RNA editing in non-human primates and nonsense mutation correction in humanized Hurler syndrome mice
    Yi, Zongyi
    Zhao, Yanxia
    Yi, Zexuan
    Zhang, Yongjian
    Tang, Gangbin
    Zhang, Xiaoxue
    Tang, Huixian
    Zhang, Wei
    Zhao, Ying
    Xu, Huayuan
    Nie, Yuyang
    Sun, Xueqing
    Xing, Lijun
    Dai, Lian
    Yuan, Pengfei
    Wei, Wensheng
    GENOME BIOLOGY, 2023, 24 (01)
  • [46] Long-Term Correction of Glycogen Storage Disease Type III by AAV-Mediated Gene Therapy Using a Dual Promoter to Express Bacterial Pullulanase
    Lim, Jeong-A
    Choi, Su Jin
    Kishnani, Priya S.
    Sun, Baodong
    MOLECULAR THERAPY, 2022, 30 (04) : 119 - 120
  • [47] In vivo AAV-mediated highly-specific transduction of Adipose Tissue in Adult Mice using microRNA Target Sequences
    Casana, E.
    Jimenez, V.
    Munoz, S.
    Jambrina, C.
    Mallol, C.
    Sacristan, V.
    Rodo, J.
    Leon, X.
    Garcia, M.
    Ribera, A.
    Bosch, F.
    HUMAN GENE THERAPY, 2015, 26 (10) : A66 - A66
  • [48] Trans-Ocular Electric Current In Vivo Enhances AAV-Mediated Retinal Transduction in Large Animal Eye After Intravitreal Vector Administration
    Song, Hongman
    Zeng, Yong
    Pasha, Sheik Pran Babu Sardar
    Bush, Ronald A.
    Vijayasarathy, Camasamudram
    Qian, Haohua
    Wei, Lisa
    Wiley, Henry E.
    Wu, Zhijian
    Sieving, Paul A.
    TRANSLATIONAL VISION SCIENCE & TECHNOLOGY, 2020, 9 (07): : 1 - 13
  • [49] Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
    Federico Mingozzi
    Katherine A. High
    Nature Reviews Genetics, 2011, 12 : 341 - 355
  • [50] Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
    Mingozzi, Federico
    High, Katherine A.
    NATURE REVIEWS GENETICS, 2011, 12 (05) : 341 - 355