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下一代生物载体平台,用于在体内输送基因组编辑剂

Delphine Leclerc¹, Michael D Siroky¹, Shannon M Miller¹

¹Department of Chemistry, The Scripps Research Institute, La Jolla, CA 92037, USA.

Current opinion in biotechnology

|December 16, 2023

概括

基因编辑CRISPR显示出对疾病的希望,但体内传递仍然是一个挑战. 本综述探讨了生物传递系统,如病毒载体和蛋白质纳米,以获得更安全,更有效的CRISPR疗法.

科学领域:

生物技术是生物技术.
分子生物学分子生物学
基因治疗基因治疗

背景情况:

基于CRISPR的基因组编辑是一种快速发展的技术,在遗传疾病,传染病和癌症中具有潜在的应用.
目前的临床试验在很大程度上仅限于ex vivo基因疗法,因为缺乏安全和有效的CRISPR组件体内输送方法.
在体内有效的CRISPR传递需要确保目标细胞转导的载体,同时最大限度地减少非目标效应,毒性和免疫反应.

研究的目的:

审查有希望的生物衍生平台,以在体内提供DNA编辑剂.
讨论这些交付平台的工程策略.
为了突出基于病毒的载体,蛋白质纳米和哺乳动物衍生的颗粒用于CRISPR传递的进步.

主要方法:

关于CRISPR传递系统近期进展的文献综述.
对生物衍生平台的分析,包括病毒载体,蛋白质纳米和哺乳动物衍生颗粒.
检查工程方法,以提高配送车辆的安全性和有效性.

主要成果:

确定基于病毒的载体,蛋白质纳米和哺乳动物衍生的颗粒作为体内CRISPR传递的有希望平台.
讨论了改进细胞转导和减少非果效应,毒性和免疫性的工程策略.
强调了这些生物衍生系统的潜力,以克服CRISPR基因疗法的当前局限性.

结论:

生物衍生交付平台为推进体内CRISPR基因组编辑提供了显著的潜力.
这些系统的进一步工程对于开发安全和有效的临床应用至关重要.
这些平台可以使CRISPR技术在超越ex vivo疗法之外得到更广泛的应用.

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