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CRISPR/Cas9 递送方法在干细胞治疗性基因编辑中的最新进展。

Recent Advances in CRISPR/Cas9 Delivery Approaches for Therapeutic Gene Editing of Stem Cells.

机构信息

Student Research Committee, Faculty of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran.

Medical Genetics Research Center, Mashhad University of Medical Sciences, Mashhad, Iran.

出版信息

Stem Cell Rev Rep. 2023 Nov;19(8):2576-2596. doi: 10.1007/s12015-023-10585-3. Epub 2023 Sep 18.

Abstract

Rapid advancement in genome editing technologies has provided new promises for treating neoplasia, cardiovascular, neurodegenerative, and monogenic disorders. Recently, the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) system has emerged as a powerful gene editing tool offering advantages, including high editing efficiency and low cost over the conventional approaches. Human pluripotent stem cells (hPSCs), with their great proliferation and differentiation potential into different cell types, have been exploited in stem cell-based therapy. The potential of hPSCs and the capabilities of CRISPR/Cas9 genome editing has been paradigm-shifting in medical genetics for over two decades. Since hPSCs are categorized as hard-to-transfect cells, there is a critical demand to develop an appropriate and effective approach for CRISPR/Cas9 delivery into these cells. This review focuses on various strategies for CRISPR/Cas9 delivery in stem cells.

摘要

基因组编辑技术的快速发展为治疗肿瘤、心血管、神经退行性和单基因疾病提供了新的希望。最近,成簇规律间隔短回文重复序列(CRISPR)/CRISPR 相关蛋白 9(Cas9)系统已成为一种强大的基因编辑工具,与传统方法相比,具有编辑效率高和成本低的优势。人类多能干细胞(hPSC)具有强大的增殖和分化潜能,可分化为不同类型的细胞,已被用于基于干细胞的治疗。hPSC 和 CRISPR/Cas9 基因组编辑的潜力在医学遗传学中已经改变了范式,超过 20 年。由于 hPSC 被归类为难转染细胞,因此迫切需要开发一种合适且有效的方法将 CRISPR/Cas9 递送到这些细胞中。本综述重点介绍了用于干细胞中 CRISPR/Cas9 递送的各种策略。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d13d/10661828/4c19277d7730/12015_2023_10585_Fig1_HTML.jpg

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