Zhang Man-Ling, Li Hong-Bin, Jin Yong
Department of Rheumatology and Immunology, The Affiliated Hospital of Inner Mongolia Medical University, Hohhot, China.
Inner Mongolia Key Laboratory for Pathogenesis and Diagnosis of Rheumatic and Autoimmune Diseases, The Affiliated Hospital of Inner Mongolia Medical University, Hohhot, China.
Front Genet. 2024 Apr 11;15:1364742. doi: 10.3389/fgene.2024.1364742. eCollection 2024.
The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) mediated Cas9 nuclease system has been extensively used for genome editing and gene modification in eukaryotic cells. CRISPR/Cas9 technology holds great potential for various applications, including the correction of genetic defects or mutations within the human genome. The application of CRISPR/Cas9 genome editing system in human disease research is anticipated to solve a multitude of intricate molecular biology challenges encountered in life science research. Here, we review the fundamental principles underlying CRISPR/Cas9 technology and its recent application in neurodegenerative diseases, cardiovascular diseases, autoimmune related diseases, and cancer, focusing on the disease modeling and gene therapy potential of CRISPR/Cas9 in these diseases. Finally, we provide an overview of the limitations and future prospects associated with employing CRISPR/Cas9 technology for diseases study and treatment.
成簇规律间隔短回文重复序列（CRISPR）介导的Cas9核酸酶系统已被广泛用于真核细胞的基因组编辑和基因修饰。CRISPR/Cas9技术在各种应用中具有巨大潜力，包括纠正人类基因组中的遗传缺陷或突变。预计CRISPR/Cas9基因组编辑系统在人类疾病研究中的应用将解决生命科学研究中遇到的众多复杂分子生物学挑战。在此，我们回顾CRISPR/Cas9技术的基本原理及其最近在神经退行性疾病、心血管疾病、自身免疫相关疾病和癌症中的应用，重点关注CRISPR/Cas9在这些疾病中的疾病建模和基因治疗潜力。最后，我们概述了将CRISPR/Cas9技术用于疾病研究和治疗的局限性及未来前景。
