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Base editing effectively prevents early-onset severe cardiomyopathy in Mybpc3 mutant mice.

作者信息

Wu Shuo, Yang Ping, Geng Zilong, Li Yige, Guo Zhizhao, Lou Yingmei, Zhang Shasha, Xiong Junhao, Hu Huan, Guo Xiaoling, Pu William T, Zhang Yan, Zhu Dan, Zhang Bing

机构信息

Key Laboratory of Systems Biomedicine, Shanghai Center for Systems Biomedicine, Department of Cardiovascular Surgery, Shanghai Chest Hospital, Engineering Research Center of Techniques and Instruments for Diagnosis and Treatment of Congenital Heart Disease, Institute of Developmental and Regenerative Medicine, Xin Hua Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai, China.

Basic Medical Research Center, the Second Affiliated Hospital and Yuying Children's Hospital of Wenzhou Medical University, Wenzhou, Zhejiang, China.

出版信息

Cell Res. 2024 Apr;34(4):327-330. doi: 10.1038/s41422-024-00930-7. Epub 2024 Feb 9.

DOI:10.1038/s41422-024-00930-7
PMID:38337022
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10978934/
Abstract
摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4aef/10978934/c34ab253ef55/41422_2024_930_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4aef/10978934/c34ab253ef55/41422_2024_930_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4aef/10978934/c34ab253ef55/41422_2024_930_Fig1_HTML.jpg

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2
Base editing correction of hypertrophic cardiomyopathy in human cardiomyocytes and humanized mice.碱基编辑纠正人类心肌细胞和人源化小鼠肥厚型心肌病。
Nat Med. 2023 Feb;29(2):401-411. doi: 10.1038/s41591-022-02176-5. Epub 2023 Feb 16.
3
Therapeutic Exon Skipping Through a CRISPR-Guided Cytidine Deaminase Rescues Dystrophic Cardiomyopathy in Vivo.
Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group.
用于基因编辑的先进递送系统：来自GenE-HumDi成本行动工作组的全面综述
Mol Ther Nucleic Acids. 2025 Jan 17;36(1):102457. doi: 10.1016/j.omtn.2025.102457. eCollection 2025 Mar 11.
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From bench to bedside: cutting-edge applications of base editing and prime editing in precision medicine.从实验室到临床：碱基编辑和引导编辑在精准医学中的前沿应用
J Transl Med. 2024 Dec 20;22(1):1133. doi: 10.1186/s12967-024-05957-3.
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Emerging CRISPR Therapies for Precision Gene Editing and Modulation in the Cardiovascular Clinic.新兴的 CRISPR 疗法在心血管临床中的精确基因编辑和调控。
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