CRISPR-Cas9 用于癌症治疗：机遇与挑战。

CRISPR-Cas9 for cancer therapy: Opportunities and challenges.

机构信息

Key Laboratory of Imaging Diagnosis and Minimally Invasive Intervention Research, The Fifth Affiliated Hospital of Wenzhou Medical University, Affiliated Lishui Hospital of Zhejiang University, The Central Hospital of Zhejiang Lishui, Lishui, 323000, China.

Department of Interventional Radiology, Shanghai St. Luke's Hospital, Shanghai, 200050, China.

出版信息

Cancer Lett. 2019 Apr 10;447:48-55. doi: 10.1016/j.canlet.2019.01.017. Epub 2019 Jan 23.

DOI:10.1016/j.canlet.2019.01.017

PMID:30684591

Abstract

Cancer is a genetic disease stemming from cumulative genetic/epigenetic aberrations. Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cas9-mediated genome editing technology has been extensively applied in various cell types and organisms, both in vitro and in vivo, for efficient gene disruption and gene modification. CRISPR-Cas9 has shown great promise for the treatment of cancer. However, despite its advantages and tremendous potential, numerous challenges, such as fitness of edited cells, editing efficiency, delivery methods and potential off-target effects, remain to be solved for completely clinical application. Here, we present the potential applications and recent advances of CRISPR-Cas9 in cancer therapy, and discuss the challenges that might be encountered in clinical applications.

摘要

癌症是一种源于累积性遗传/表观遗传异常的基因疾病。成簇规律间隔短回文重复序列（CRISPR）-Cas9 介导的基因组编辑技术已广泛应用于体外和体内的各种细胞类型和生物体中，用于高效的基因敲除和基因修饰。CRISPR-Cas9 在癌症治疗方面显示出巨大的潜力。然而，尽管具有优势和巨大的潜力，但仍有许多挑战需要解决，例如编辑细胞的适应性、编辑效率、递送方法和潜在的脱靶效应，才能完全应用于临床。在这里，我们介绍了 CRISPR-Cas9 在癌症治疗中的潜在应用和最新进展，并讨论了在临床应用中可能遇到的挑战。