非病毒CRISPR/Cas9系统载体的研究进展
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摘要
近年来,规律成簇的间隔短回文重复序列(clustered regularly interspaced short palindromic repeats sequences, CRISPR)/CRISPR相关蛋白9(CRISPR associated protein 9, Cas9)基因编辑系统已经成为生物医学基础研究中最强大的技术之一.但是如何将CRISPR/Cas9系统高效地递送至靶器官或细胞仍是一个巨大的挑战.随着非病毒载体的快速发展,基于脂质体、聚合物、无机纳米粒子等化学方法的纳米载体已经显现出巨大的应用潜力.本文首先对CRISPR/Cas9系统在疾病建模和疾病治疗中的应用潜力以及其局限性进行了阐述,然后分析了以质粒DNA、mRNA或蛋白质形式递送CRISPR/Cas9系统的优缺点并详细概述了已经出现的非病毒载体,最后总结了当前非病毒载体在递送CRISPR/Cas9系统过程中面临的关键障碍,并提出了有望克服这些障碍的策略.
In recent years, clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR associated protein 9(Cas9) system has become one of the most powerful technologies in biomedical research and shown great potentials in the establishment of disease models as well as the development of new treatments. However, efficient delivery of CRISPR/Cas9 systems to target organs and cells remains challenging. With the rapid development of nonviral vectors, nanocarriers based on liposomes, polymers, and inorganic nanoparticles have shown great potentials for the delivery of CRISPR/Cas9 systems. In this review, we first summarize the potential of the CRISPR/Cas9 system in disease modeling and disease treatment, as well as the limitations. The advantages and disadvantages of delivering the CRISPR/Cas9 system in the form of plasmid DNA, mRNA or protein are then analyzed and the non-viral vectors that have emerged are reviewed. Finally, we summarize the key barriers currently faced by non-viral vectors in the delivery of CRISPR/Cas9 systems and propose strategies that are expected to overcome them.
In recent years, clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR associated protein 9(Cas9) system has become one of the most powerful technologies in biomedical research and shown great potentials in the establishment of disease models as well as the development of new treatments. However, efficient delivery of CRISPR/Cas9 systems to target organs and cells remains challenging. With the rapid development of nonviral vectors, nanocarriers based on liposomes, polymers, and inorganic nanoparticles have shown great potentials for the delivery of CRISPR/Cas9 systems. In this review, we first summarize the potential of the CRISPR/Cas9 system in disease modeling and disease treatment, as well as the limitations. The advantages and disadvantages of delivering the CRISPR/Cas9 system in the form of plasmid DNA, mRNA or protein are then analyzed and the non-viral vectors that have emerged are reviewed. Finally, we summarize the key barriers currently faced by non-viral vectors in the delivery of CRISPR/Cas9 systems and propose strategies that are expected to overcome them.
引文
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