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Updated: Jan 18, 2026

CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy
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Published on: March 12, 2018

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推进基因治疗基尿症:从精确编辑到临床翻译

Inseon Yu1, Jaemin Jeong1

  • 1Department of Biohealth Convergence, College of Science and Convergence Technology, Seoul Women's University, Seoul 01797, Republic of Korea.

International journal of molecular sciences
|September 13, 2025
PubMed
概括

基因疗法为遗传性疾病基尿症 (PKU) 提供了新的希望. 腺基编辑和病毒基因转移在临床前研究中显示出治疗PKU的前景.

关键词:
基尿症 (PKU) 是一种腺相关病毒的病毒.动物模型动物模型基础编辑 基础编辑基因治疗的基因疗法

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