Advancements in CRISPR/Cas systems for disease treatment.
10.1016/j.apsb.2025.05.007
- Author:
Yangsong XU
1
;
Hao LE
1
;
Qinjie WU
1
;
Ning WANG
1
;
Changyang GONG
1
Author Information
1. Department of Biotherapy, Cancer Center and State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, China.
- Publication Type:Review
- Keywords:
CRISPR/Cas classification;
CRISPR/Cas9;
Deliver;
Disease treatment;
Gene editing
- From:
Acta Pharmaceutica Sinica B
2025;15(6):2818-2844
- CountryChina
- Language:English
-
Abstract:
The clustered regularly interspaced short palindromic repeats (CRISPR)-Cas (CRISPR-associated proteins) is an adaptive immune system present in most bacteria and archaea, protecting them from infection by exogenous genetic elements. Due to its simplicity, cost-effectiveness, and precise gene editing capabilities, CRISPR/Cas technology has emerged as a promising tool for treating diseases. The continuous refinement of derivative systems has further broadened its scope in disease treatment. Nevertheless, the heterogeneous physiopathological nature of diseases and variations in disease onset sites pose significant challenges for in vivo applications of CRISPR systems. The efficiency of CRISPR systems in disease treatment is directly influenced by the performance of the delivery system. Additionally, concerns such as off-target effects present crucial hurdles in the clinical implementation of CRISPR systems. This review provides a comprehensive overview of the development of CRISPR systems, vector technologies, and their applications in disease treatment, while also addressing the challenges encountered in clinical settings. Furthermore, future research directions are outlined to pave the way for advancements in CRISPR-based therapies.
