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对基于CRISPR的罕见疾病临床试验的当前状态进行全面审查

Amneet Kaur Badwal¹, Sushma Singh¹

¹Department of Biotechnology, National Institute of Pharmaceutical Education and Research, S.A.S. Nagar, Mohali 160062, Punjab, India.

International journal of biological macromolecules

|July 26, 2024

概括

聚类定期间隔的简短巴林德罗姆重复 (CRISPR) 基因疗法在治疗诸如状细胞贫血等罕见疾病方面表现有前途. 目前正在进行的临床试验评估基于CRISPR/Cas的治疗方法,重点关注疗效,安全性和伦理考虑.

科学领域:

遗传学和基因组学在
生物技术是生物技术.
医学研究医学研究

背景情况:

罕见疾病影响着全球大量人口,需要创新的治疗策略.
集群定期间隔的简短的Palindromic重复 (CRISPR) 和Cas蛋白为罕见的遗传疾病提供先进的基因疗法.
目标罕见疾病的例子包括遗传性转基因氨基粉症,遗传性血管,杜申肌肉发育不良,雷特综合征和像亨廷顿病这样的神经疾病.

研究的目的:

审查目前正在临床试验中的罕见疾病的基于CRISPR/Cas的疗法.
讨论与CRISPR/Cas基因编辑疗法相关的传递方法,疗效,安全性,约束和伦理问题.

主要方法:

审查关于CRISPR/Cas在罕见疾病治疗中的应用现有文献.
对基因疗法传递系统 (矢量介导和非矢量介导) 的分析.
检查临床试验数据和CRISPR/Cas疗法的报告结果.

主要成果:

克里斯普尔/Cas9已经证明在治疗β-血病和状细胞疾病等疾病方面具有有效性.
基因疗法输送方法根据细胞类型,表达需求和遗传物质大小而有所不同.
复原病毒和病毒载体 (例如,BB305) 用于ex vivo基因疗法,并考虑整合和表达水平.

结论:

基于CRISPR/Cas的疗法正在通过各种罕见疾病的临床试验取得进展.
这些基因组编辑工具的未来应用取决于证明其强大的有效性和安全性.
解决制约因素和伦理考虑对于成功实施CRISPR/Cas基因疗法至关重要.

关键词:

这就是CRISPR/Cas9的作用.临床试验中的临床试验.基因组编辑基因组编辑罕见疾病是一种罕见的疾病.状细胞疾病是一种状细胞疾病.在β-Thalassemia中,

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