CRISPR-Cas9基因编辑技术及其在人类治疗中的现有作用。
CRISPR-Cas9 base editors and their current role in human therapeutics.
发表日期:2023 Mar
作者:
Walker S Lahr, Christopher J Sipe, Joseph G Skeate, Beau R Webber, Branden S Moriarity
来源:
CYTOTHERAPY
摘要:
一直在持续进展的是创造更加高效和有用的CRISPR-Cas9基因组改良分子工具。这篇综述主要关注最近用基因编辑酶(BEs)进行临床和研究目的的文章。由于CRISPR-Cas9 BEs的高效性和广泛适用于基因修正和破坏,所以这是一个有用的系统。此外,与其他CRISPR-Cas9系统相比,基因编辑被建议作为一种更安全的方法,因为在多基因敲除过程中限制了双链断裂,且不需要毒性的DNA供体分子进行基因修正。因此,许多行业和学术团体正在开发基于基因编辑酶的临床应用,如癌症免疫疗法和基因治疗。本文将重点探讨现在和未来基于体内BE递送的应用及其临床应用。
Consistent progress has been made to create more efficient and useful CRISPR-Cas9-based molecular toolsfor genomic modification.This review focuses on recent articles that have employed base editors (BEs) for both clinical and research purposes.CRISPR-Cas9 BEs are a useful system because of their highefficiency and broad applicability to gene correction and disruption. In addition, base editing has beensuggested as a safer approach than other CRISPR-Cas9-based systems, as it limits double-strand breaksduring multiplex gene knockout and does not require a toxic DNA donor molecule for genetic correction.As such, numerous industry and academic groups are currently developing base editing strategies withclinical applications in cancer immunotherapy and gene therapy, which this review will discuss, with a focuson current and future applications of in vivo BE delivery.Published by Elsevier Inc.