基于CRISPR的基编辑作为罕见单基性疾病的治疗策略

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Updated: May 11, 2025

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Published on: February 17, 2023

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Júlia-Jié Cabré-Romans1,2, Raquel Cuella-Martin1,2

  • 1Department of Human Genetics, McGill University, Montreal, QC, Canada.

Frontiers in genome editing
|April 17, 2025
PubMed
概括

基因编辑疗法,特别是CRISPR基因编辑,通过精确纠正基因突变,为罕见的单一性疾病提供了有希望的单一治疗方法. 这项技术有可能解决这些低流行病的重大未满足的医疗需求.

关键词:
基于CRISPR的治疗方法基础编辑 基础编辑基因治疗的基因疗法突变纠正 突变纠正罕见的单一性疾病罕见的单一性疾病.

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