Chodnekar Swarali Yatin, Tsetskhladze Zurab
Faculty of Medicine, University Geomedi, 3, King Solomon Street, Tbilisi 0114, Georgia.
Oxf Open Immunol. 2024 Nov 21;5(1):iqae013. doi: 10.1093/oxfimm/iqae013. eCollection 2024.
To consolidate clinical trials that utilized the CRISPR technology to synthesise cures for various genetic diseases as a means to provide a window into the progress made so far while paving the way forward for future research and practices. Systematic review (PROSPERO CRD42023479511). Trials from seven databases' (ClinicalTrials.gov, European Union Clinical Trials Registry, ISRCTN registry, ICTRP/trialsearch.who.int, ChiCTR.org.cn, Clinical Trial Registry India, and Cochrane Library/Trials) inception to 9 March 2024, were considered. Exclusion criteria were unrelated, duplicated, non-English, unavailable full texts, diagnostic studies, correlational studies, observational studies, abstract-only papers, reviews or conference papers. Included studies were appraised using the ten-item CASP tool to assess methodological quality. The review identified 82 RCTs utilizing CRISPR and revealed four main themes: Diseases targeted, Countries of Clinical trials, Type of interventions, and Trial trends over the years. Geographically, the United States and China lead in the number of CRISPR clinical trials, followed by the European Union. However, Africa, Asia, and South America have very few trials. Among disease classes, cancer is the most prevalent focus with 39 studies, followed by monogenetic blood diseases, like Thalassemia and sickle cell anaemia. The biological agent CTX001 and Cyclophosphamide each feature in 11 studies. The peak year for clinical trials was 2018, marked by a significant increase with 16 studies conducted. Despite conducting a comprehensive search, the majority of trials were concentrated in the United States and China. Additionally, potential oversights due to vague titles, English-only studies, and indexing issues may have occurred. Nonetheless, by incorporating data from seven distinct databases, this review significantly contributes to understanding CRISPR's utilization in therapeutic clinical trials, paving the way for future research directions. The review underscores the burgeoning interest in CRISPR-based interventions. Current trials barely tap CRISPR's potential for treating genetic diseases.
整合利用CRISPR技术合成各种遗传疾病治疗方法的临床试验，以此为迄今取得的进展提供一个窗口，同时为未来的研究和实践铺平道路。系统评价（PROSPERO CRD42023479511）。考虑了来自七个数据库（ClinicalTrials.gov、欧盟临床试验注册中心、ISRCTN注册中心、ICTRP/trialsearch.who.int、ChiCTR.org.cn、印度临床试验注册中心和Cochrane图书馆/试验）从创建到2024年3月9日的试验。排除标准为不相关、重复、非英文、无法获取全文、诊断研究、相关性研究、观察性研究、仅摘要论文、综述或会议论文。纳入的研究使用十项CASP工具进行评估，以评估方法学质量。该评价确定了82项利用CRISPR的随机对照试验，并揭示了四个主要主题：靶向疾病、临床试验国家、干预类型和多年来的试验趋势。从地理上看，美国和中国在CRISPR临床试验数量上领先，其次是欧盟。然而，非洲、亚洲和南美洲的试验很少。在疾病类别中，癌症是最普遍的重点，有39项研究，其次是单基因血液疾病，如地中海贫血和镰状细胞贫血。生物制剂CTX001和环磷酰胺各有11项研究。临床试验的高峰年份是2018年，有16项研究显著增加。尽管进行了全面搜索，但大多数试验集中在美国和中国。此外，由于标题模糊、仅英文研究和索引问题可能存在潜在疏漏。尽管如此，通过纳入来自七个不同数据库的数据，本评价对理解CRISPR在治疗性临床试验中的应用做出了重大贡献，为未来的研究方向铺平了道路。该评价强调了对基于CRISPR的干预措施的迅速增长的兴趣。目前的试验几乎没有挖掘出CRISPR治疗遗传疾病的潜力。
