Department of Bioengineering, Clemson University, Clemson, SC 29634, USA.
College of Medicine, Medical University of South Carolina, Charleston, SC 29425, USA.
Genes (Basel). 2020 Sep 23;11(10):1113. doi: 10.3390/genes11101113.
Impressive therapeutic advances have been possible through the advent of zinc-finger nucleases and transcription activator-like effector nucleases. However, discovery of the more efficient and highly tailorable clustered regularly interspaced short palindromic repeats (CRISPR) and associated proteins (Cas9) has provided unprecedented gene-editing capabilities for treatment of various inherited and acquired diseases. Despite recent clinical trials, a major barrier for therapeutic gene editing is the absence of safe and effective methods for local and systemic delivery of gene-editing reagents. In this review, we elaborate on the challenges and provide practical considerations for improving gene editing. Specifically, we highlight issues associated with delivery of gene-editing tools into clinically relevant cells.
通过锌指核酸酶和转录激活因子样效应物核酸酶的出现，实现了令人瞩目的治疗进展。然而，更高效和高度可定制的成簇规律间隔短回文重复序列 (CRISPR) 及其相关蛋白 (Cas9) 的发现，为治疗各种遗传性和获得性疾病提供了前所未有的基因编辑能力。尽管最近进行了临床试验，但治疗性基因编辑的一个主要障碍是缺乏安全有效的局部和全身基因编辑试剂传递方法。在这篇综述中，我们详细阐述了面临的挑战，并提供了改善基因编辑的实用考虑因素。具体来说，我们强调了与将基因编辑工具递送到临床相关细胞相关的问题。
