从长椅到床边:基因组编辑应用的伦理和临床最佳实践
María Ortiz-Bueno1, Federica Zinghirino2, Pilar Puig Serra3
1GENYO, Centre for Genomics and Oncological Research: Pfizer, University of Granada, 18016 Granada, Spain.
International journal of molecular sciences
|February 13, 2026
概括
基因组编辑 (GE) 通过精确改变DNA提供了新的医学治疗方法. 负责任的临床应用需要严格关注安全,伦理和监管标准,以确保患者的福祉.
科学领域:
- 生物技术和生物医学科学 生物技术和生物医学科学
- 遗传学和基因组学 遗传学和基因组学
- 医学伦理和法规 医学伦理和法规
背景情况:
- 基因组编辑 (GE) 技术,包括指核酶 (ZFNs),转录激活器样效应核酶 (TALENs) 和基于定期间隔的短平行列重复 (CRISPR) 的系统,已经彻底改变了医学.
- 目前,CRISPR系统是最容易获得,最多功能,最广泛采用的GE技术.
- 尽管GE对遗传性和获得性疾病具有治疗潜力,但临床翻译面临着重大的安全,伦理和监管障碍.
研究的目的:
- 审查当前的科学文献和监管机构对基因组编辑负责任的临床应用的建议.
- 要突出关键的考虑患者安全,道德实践,和监管合规在基因转基因疗法.
- 提供与GE医疗用途相关的技术和监管挑战的概述.
主要方法:
- 关于基因组编辑的发表科学研究的文献综述.
- 分析FDA和EMA等监管机构的指导文件和建议.
- 综合有关临床GE安全性,伦理考虑和监管框架的信息.
主要成果:
- 转基因技术提供了有前途的治疗途径,但也带来了诸如非目标突变和意外细胞修饰等挑战.
- 监管机构强调严格的临床前检测,患者监测,知情同意和遵守法律标准.
- 协调的安全标准和生物伦理考虑对于GE的临床进步至关重要.
结论:
- 基因组编辑的负责任的临床应用需要采用综合方法,将科学进步与严格的安全协议,道德监督和监管合规相结合.
- 应对技术挑战和导航监管环境对于实现GE的全部治疗潜力至关重要.
- 在整个基因组编辑疗法的开发和部署过程中,患者安全和道德实践必须保持至关重要的地位.
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