First in vivo base-editing trial shows promise

被引：4
|
作者：
Han, Renzhi [1 ]
机构：
[1] Indiana Univ Sch Med, Herman B Wells Ctr Pediat Res, Dept Pediat, 980 W Walnut St,Joseph E Walther Hall R3 C118, Indianapolis, IN 46202 USA
来源：
MOLECULAR THERAPY | 2024年 / 32卷 / 01期
关键词：
GENOMIC DNA; PCSK9; LDL;
D O I：
10.1016/j.ymthe.2023.12.001
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：
引用
收藏
页码：1 / 2
页数：2
相关论文
共 50 条
  • [21] Creation of herbicide-resistance in allotetraploid peanut using CRISPR/Cas9-meditated cytosine base-editing
    Shi, Lei
    Li, Xiaona
    Xue, Lulu
    Zhang, Jin
    Huang, Bingyan
    Sun, Ziqi
    Zhang, Zhongxin
    Dai, Xiaodong
    Han, Suoyi
    Dong, Wenzhao
    Zhang, Xinyou
    PLANT BIOTECHNOLOGY JOURNAL, 2023, 21 (10) : 1923 - 1925
  • [22] In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates
    Musunuru, Kiran
    Chadwick, Alexandra C.
    Mizoguchi, Taiji
    Garcia, Sara P.
    DeNizio, Jamie E.
    Reiss, Caroline W.
    Wang, Kui
    Iyer, Sowmya
    Dutta, Chaitali
    Clendaniel, Victoria
    Amaonye, Michael
    Beach, Aaron
    Berth, Kathleen
    Biswas, Souvik
    Braun, Maurine C.
    Chen, Huei-Mei
    Colace, Thomas, V
    Ganey, John D.
    Gangopadhyay, Soumyashree A.
    Garrity, Ryan
    Kasiewicz, Lisa N.
    Lavoie, Jennifer
    Madsen, James A.
    Matsumoto, Yuri
    Mazzola, Anne Marie
    Nasrullah, Yusuf S.
    Nneji, Joseph
    Ren, Huilan
    Sanjeev, Athul
    Shay, Madeleine
    Stahley, Mary R.
    Fan, Steven H. Y.
    Tam, Ying K.
    Gaudelli, Nicole M.
    Ciaramella, Giuseppe
    Stolz, Leslie E.
    Malyala, Padma
    Cheng, Christopher J.
    Rajeev, Kallanthottathil G.
    Rohde, Ellen
    Bellinger, Andrew M.
    Kathiresan, Sekar
    NATURE, 2021, 593 (7859) : 429 - +
  • [23] Jumping on base editing to repair the diseased cardiovascular system in vivo
    Gladka, Monika M.
    Baker, Andrew H.
    CARDIOVASCULAR RESEARCH, 2021, 117 (04) : E46 - E48
  • [24] In vivo base editing of post-mitotic sensory cells
    Yeh, Wei-Hsi
    Chiang, Hao
    Rees, Holly A.
    Edge, Albert S. B.
    Liu, David R.
    NATURE COMMUNICATIONS, 2018, 9
  • [25] Efficient precise in vivo base editing in adult dystrophic mice
    Xu, Li
    Zhang, Chen
    Li, Haiwen
    Wang, Peipei
    Gao, Yandi
    Mokadam, Nahush A.
    Ma, Jianjie
    Arnold, W. David
    Han, Renzhi
    NATURE COMMUNICATIONS, 2021, 12 (01)
  • [26] In vivo application of base and prime editing to treat inherited retinal diseases
    Jo, Dong Hyun
    Bae, Sangsu
    Kim, Hyongbum Henry
    Kim, Jin-Soo
    Kim, Jeong Hun
    PROGRESS IN RETINAL AND EYE RESEARCH, 2023, 94
  • [27] In vivo adenine base editing rescues adrenoleukodystrophy in a humanized mouse model
    Gopalappa, Ramu
    Lee, MinYoung
    Kim, Globinna
    Jung, Eul Sik
    Lee, Hanahrae
    Hwang, Hye-Yeon
    Lee, Jong Geol
    Kim, Su Jung
    Yoo, Hyun Ju
    Sung, Young Hoon
    Kim, Daesik
    Baek, In-Jeoung
    Kim, Hyongbum Henry
    MOLECULAR THERAPY, 2024, 32 (07) : 2190 - 2206
  • [28] In vivo base editing rescues primary hyperoxaluria type 1 in rats
    Chen, Zhoutong
    Zhang, Dexin
    Zheng, Rui
    Yang, Lei
    Huo, Yanan
    Zhang, Dan
    Fang, Xiaoliang
    Li, Yueyan
    Xu, Guofeng
    Li, Dali
    Geng, Hongquan
    KIDNEY INTERNATIONAL, 2024, 105 (03) : 496 - 507
  • [29] Minimizing the ratio of ionizable lipid in lipid nanoparticles for in vivo base editing
    Chen, Qiubing
    Wang, Xuebin
    Zhang, Yizhou
    Tian, Ming
    Duan, Junyi
    Zhang, Ying
    Yin, Hao
    NATIONAL SCIENCE REVIEW, 2024, 11 (06)
  • [30] Lipid nanoparticle-mediated base-editing of the Hao1 gene achieves sustainable primary hyperoxaluria type 1 therapy in rats
    Zhang, Dexin
    Zheng, Rui
    Chen, Zhoutong
    Wang, Liren
    Chen, Xi
    Yang, Lei
    Huo, Yanan
    Yin, Shuming
    Zhang, Dan
    Huang, Jiaxin
    Cui, Xingang
    Li, Dali
    Geng, Hongquan
    SCIENCE CHINA-LIFE SCIENCES, 2024, 67 (12) : 2575 - 2586
12345