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血症中的CRISPR:全球研究趋势分析

Muhammad Saboor^1,2, Maryam Jasem Alblooshi¹, Alreem Adel Alkaabi¹

¹Department of Medical Laboratory Sciences, College of Health Sciences, University of Sharjah, Sharjah, United Arab Emirates.

|March 6, 2026

概括

基因编辑CRISPR显示出治疗β-血病的前景,主要来自美国和中国的研究. 两个主要策略,HBB基因校正和BCL11A抑制导致Casgevy批准,正在推进临床翻译.

科学领域:

生物技术是生物技术.
遗传学是一个遗传学.
血液学血液学血液学

背景情况:

β-thalassemia是一种常见的遗传性血液疾病,影响β-环球蛋白链.
聚类正规间隔短平行体重复 (CRISPR) 基因组编辑系统提供了一个潜在的治疗方法.
图书统计分析对于理解新兴领域的全球研究环境至关重要.

研究的目的:

绘制基于CRISPR的β-thalassemia研究的全球研究趋势.
确定关键的研究领域,合作和领先的机构.
审查CRISPR治疗策略及其转化进展.

主要方法:

从最初到现在,对Scopus数据库文章 (原始和复习) 的图书统计分析.
搜索术语包括"贝塔血症"和"CRISPR"/"基因编辑"变体.
VOSviewer软件用于出版物,作者和关键字的网络可视化.

主要成果:

基于CRISPR的血病研究显著增长,美国和中国是主要贡献者.
确定了两个主要的治疗策略:直接的HBB基因校正和BCL11A抑制用于胎儿血红蛋白重新激活.
基于BCL11A抑制的Exagamglogene autotemcel (Casgevy) 获得了监管部门的批准.

结论:

对血症的CRISPR研究正在从基础科学向临床应用过渡.
国际合作广泛,推动基因编辑疗法的进步.
未来的实施需要生物处理,降低成本和适应性医疗保健模型的进步.

关键词:

克里斯普尔是什么意思?克里斯普尔是什么意思?泰拉西米亚泰拉西米亚文献学分析文献学分析基因编辑基因编辑血红蛋白血红蛋白是一个

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