Inflammation and Immune Mediated Diseases Laboratory of Anhui Province, Anhui Institute of Innovative Drugs, School of Pharmacy, Anhui Medical University, 81 Meishan Road, Hefei 230032, Anhui Province, China.
Institute for Liver Diseases of Anhui Medical University, Anhui Medical University, Hefei 230032, China.
Int J Biol Sci. 2020 Jun 1;16(13):2283-2295. doi: 10.7150/ijbs.33481. eCollection 2020.
Liver diseases are one of the leading causes of mortality in the world, mainly caused by different etiological agents, alcohol consumption, viruses, drug intoxication, and malnutrition. The maturation of gene therapy has heralded new avenues for developing effective interventions for these diseases. Derived from a remarkable microbial defense system, clustered regularly interspaced short palindromic repeats/CRISPR-associated proteins 9 system (CRISPR/Cas9 system) is driving innovative applications from basic biology to biotechnology and medicine. Recently, the mutagenic function of CRISPR/Cas9 system has been widely adopted for genome and disease research. In this review, we describe the development and applications of CRISPR/Cas9 system on liver diseases for research or translational applications, while highlighting challenges as well as future avenues for innovation.
肝脏疾病是世界范围内主要的致死病因之一，主要由不同的病因、酒精摄入、病毒、药物中毒和营养不良引起。基因治疗的成熟为这些疾病的有效干预措施的开发开辟了新的途径。来源于一个非凡的微生物防御系统，成簇规律间隔短回文重复序列/CRISPR 相关蛋白 9 系统（CRISPR/Cas9 系统）正在推动从基础生物学到生物技术和医学的创新应用。最近，CRISPR/Cas9 系统的诱变功能已被广泛应用于基因组和疾病研究。在这篇综述中，我们描述了 CRISPR/Cas9 系统在肝脏疾病的研究或转化应用方面的发展和应用，同时强调了挑战以及未来的创新途径。
