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将治疗性基因组编辑引入全球临床试验和医学领域
Kiran Musunuru1, Fyodor Urnov2
1Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
The CRISPR journal
|May 21, 2025
概括
克里斯普尔基因编辑疗法面临的挑战是从研究转向患者治疗. 专家们回顾了这个革命性的领域二十年的进步和未来的创新.
科学领域:
- 遗传学和基因组学 在
- 生物技术是生物技术.
- 分子生物学分子生物学
背景情况:
- 在过去的20年里,CRISPR基因编辑技术取得了显著的进步.
- 基于CRISPR的疗法的临床转化提出了从早期研究到患者剂量管理的复杂挑战.
研究的目的:
- 为开发基于CRISPR的疗法提供洞察力.
- 为了反思过去20年来基因组编辑领域的进展和挑战.
- 讨论当前的创新环境和CRISPR疗法的未来方向.
主要方法:
- 专家采访格式 专家采访格式.
- 在CRISPR基因编辑中讨论非临床和临床程序.
- 分析该领域的现状和未来前景.
主要成果:
- 该领域在推进CRISPR疗法方面经历了成功和挫折.
- 在目前的基因组编辑领域,正在发生重大创新.
- 未来的方向包括持续的研究和临床翻译.
结论:
- 从CRISPR发现到患者批准的路径是复杂的.
- 持续的创新和对过去经验的反思对于基因组编辑疗法的未来进展至关重要.
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