通过CRISPR/Cas核糖核蛋白复合物在包膜病毒衍生颗粒中的运输进行基因编辑。

IF 3.9 3区 医学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Human gene therapy Pub Date : 2024-09-01 Epub Date: 2024-08-27 DOI:10.1089/hum.2024.105
Jacob Hørlück Janns, Jacob Giehm Mikkelsen
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引用次数: 0

摘要

下一代 CRISPR/Cas 基因编辑工具(如碱基和质粒编辑)的发明,为纠正导致疾病的基因变异带来了在患者体内使用的希望,从而实现更广泛的临床转化。为了实现这一潜力,我们亟需能将基因编辑工具包安全有效地送入特定细胞群或组织的运载工具。在这里,我们介绍了开发包膜逆转录病毒衍生颗粒,作为由 Cas9 衍生编辑蛋白和单个引导 RNA 组成的 "即用型 "核糖核蛋白复合物的载体。我们提出了将病毒用于细胞靶向蛋白递送的论点,并描述了经过长达十年的开发后的现状。病毒已经在原代细胞(包括 T 细胞和造血干细胞)和体内靶向组织(包括小鼠视网膜、肝脏和大脑)中显示出有效的编辑效果。新的证据表明,病毒衍生的工程纳米粒子既可用于基础编辑,也可用于主要编辑,这似乎萌发了进一步开发和大规模生产此类粒子用于治疗应用的希望。
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Gene Editing by Ferrying of CRISPR/Cas Ribonucleoprotein Complexes in Enveloped Virus-Derived Particles.

The invention of next-generation CRISPR/Cas gene editing tools, like base and prime editing, for correction of gene variants causing disease, has created hope for in vivo use in patients leading to wider clinical translation. To realize this potential, delivery vehicles that can ferry gene editing tool kits safely and effectively into specific cell populations or tissues are in great demand. In this review, we describe the development of enveloped retrovirus-derived particles as carriers of "ready-to-work" ribonucleoprotein complexes consisting of Cas9-derived editor proteins and single guide RNAs. We present arguments for adapting viruses for cell-targeted protein delivery and describe the status after a decade-long development period, which has already shown effective editing in primary cells, including T cells and hematopoietic stem cells, and in tissues targeted in vivo, including mouse retina, liver, and brain. Emerging evidence has demonstrated that engineered virus-derived nanoparticles can accommodate both base and prime editors and seems to fertilize a sprouting hope that such particles can be further developed and produced in large scale for therapeutic applications.

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来源期刊
Human gene therapy
Human gene therapy 医学-生物工程与应用微生物
CiteScore
6.50
自引率
4.80%
发文量
131
审稿时长
4-8 weeks
期刊介绍: Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases.
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