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通过脂质纳米粒子提供基于核酸的基因组编辑平台:临床应用

Razan Masarwy¹, Lior Stotsky-Oterin², Aviad Elisha¹

¹Laboratory of Precision Nanomedicine, The Shmunis School of Biomedicine and Cancer Research, George S. Wise Faculty of Life Sciences, Tel Aviv University, Tel Aviv, Israel; Department of Materials Sciences and Engineering, Iby and Aladar Fleischman Faculty of Engineering, Tel Aviv University, Tel Aviv, Israel; Center for Nanoscience and Nanotechnology, Tel Aviv University, Tel Aviv, Israel; School of Medicine, Tel Aviv University, Tel Aviv, Israel.

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概括

脂质纳米粒子 (LNP) 为CRISPR基因编辑疗法提供了一个有前途的输送方法. 优化LNP向对于癌症和遗传疾病等疾病的有效体内治疗至关重要.

科学领域:

生物技术是生物技术.
基因治疗基因治疗
纳米医学是一种纳米医学.

背景情况:

克里斯普尔/卡斯技术对遗传疾病和癌症具有显著的治疗潜力.
对CRISPR/Cas系统来说,有效的in-vivo基因治疗仍然是一个挑战.
脂质纳米粒子 (LNP) 是FDA批准的RNA输送,可以封装CRISPR/Cas组件.

研究的目的:

审查使用LNP封装的基于CRISPR的技术用于治疗应用的进展和挑战.
通过LNP评估改善体内向和传递CRISPR/Cas组件的策略.
突出LNP提供的基础和原始编辑技术的潜力,以实现精确的基因修饰.

主要方法:

对CRISPR/Cas交付LNP配方的当前文献的审查.
分析提高LNP特异性和准in-vivo的策略.
在LNP交付的背景下评估基础和主要编辑技术.

主要成果:

简单的LNP可以有效地封装大型mRNA分子,并引导CRISPR/Cas.的RNA.
在体内目标准确性仍然是基于LNP的CRISPR交付的关键挑战.
对LNP结构的修改和向配体的加入可以提高特异性.
基础和主要编辑提供精确的基因修饰,没有双链断裂.

结论:

用LNP封装的CRISPR技术在体内基因治疗方面显示出前景.
进一步优化LNP向对于临床转化至关重要.
通过有针对性的LNP提供主要编辑为各种疾病提供了安全和精确的治疗方法.

关键词:

基础编辑器基础编辑器克里斯普尔 Cas9 案例基因编辑基因编辑基因药物基因药物基因药物脂质纳米颗粒的使用方法总编辑总编辑总编辑

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相关概念视频

CRISPR

CRISPR

Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...