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Updated: Jun 25, 2025

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最近的治疗性基因编辑应用在遗传疾病中的应用

Eric Deneault1

  • 1Regulatory Research Division, Centre for Oncology, Radiopharmaceuticals and Research, Biologic and Radiopharmaceutical Drugs Directorate, Health Products and Food Branch, Health Canada, Ottawa, ON K1A 0K9, Canada.

Current issues in molecular biology
|May 24, 2024
PubMed
概括

治疗性基因编辑,特别是CRISPR-Cas9,基因编辑和主要编辑,为遗传疾病提供精确的基因组修改. 传递系统的创新和新的CRISPR变体提高了精准医学应用的有效性和安全性.

关键词:
克里斯普尔是什么意思?克里斯普尔是什么意思?基因编辑 基因编辑遗传疾病 遗传疾病远离目标 - 远离目标

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