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.

中文翻译：

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CRISPR/Cas9基因组编辑技术在人类疾病建模和基因治疗中的应用与展望

成簇规则间隔短回文重复序列 (CRISPR) 介导的 Cas9 核酸酶系统已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的潜力，包括纠正人类基因组内的遗传缺陷或突变。 CRISPR/Cas9基因组编辑系统在人类疾病研究中的应用有望解决生命科学研究中遇到的众多复杂的分子生物学挑战。在此，我们回顾了CRISPR/Cas9技术的基本原理及其在神经退行性疾病、心血管疾病、自身免疫相关疾病和癌症中的最新应用，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，我们概述了采用 CRISPR/Cas9 技术进行疾病研究和治疗的局限性和未来前景。