Zhang Songyang, Wang Yidi, Mao Dezhi, Wang Yue, Zhang Hong, Pan Yihan, Wang Yuezeng, Teng Shuzhi, Huang Ping
The Key Laboratory of Pathobiology, Ministry of Education, Norman Bethune College of Medicine, Jilin University, Changchun, China.
The Third Affiliated Hospital of Jilin University, Changchun, China.
Front Med (Lausanne). 2023 Nov 10;10:1292452. doi: 10.3389/fmed.2023.1292452. eCollection 2023.
The CRISPR/Cas9 system is a powerful genome editing tool that has made enormous impacts on next-generation molecular diagnostics and therapeutics, especially for genetic disorders that traditional therapies cannot cure. Currently, CRISPR-based gene editing is widely applied in basic, preclinical, and clinical studies. In this review, we attempt to identify trends in clinical studies involving CRISPR techniques to gain insights into the improvement and contribution of CRISPR/Cas technologies compared to traditional modified modalities. The review of clinical trials is focused on the applications of the CRISPR/Cas systems in the treatment of cancer, hematological, endocrine, and immune system diseases, as well as in diagnostics. The scientific basis underlined is analyzed. In addition, the challenges of CRISPR application in disease therapies and recent advances that expand and improve CRISPR applications in precision medicine are discussed.
CRISPR/Cas9系统是一种强大的基因组编辑工具,对下一代分子诊断和治疗产生了巨大影响,特别是对于传统疗法无法治愈的遗传性疾病。目前,基于CRISPR的基因编辑广泛应用于基础、临床前和临床研究。在本综述中,我们试图确定涉及CRISPR技术的临床研究趋势,以深入了解与传统修饰方式相比,CRISPR/Cas技术的改进和贡献。对临床试验的综述集中于CRISPR/Cas系统在癌症、血液学、内分泌和免疫系统疾病治疗以及诊断中的应用。分析了其背后的科学依据。此外,还讨论了CRISPR在疾病治疗应用中的挑战以及在精准医学中扩展和改进CRISPR应用的最新进展。