Could targeted gene insertion of factor 9 be a potential durable treatment for Hemophilia B?

IF 2.1 4区 医学 Q2 HEMATOLOGY Expert Review of Hematology Pub Date : 2025-02-01 Epub Date: 2025-01-27 DOI:10.1080/17474086.2025.2459253
Giancarlo Castaman, Mirko Pinotti
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靶向基因插入因子9能否成为B型血友病的潜在持久治疗方法?
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来源期刊
CiteScore
4.70
自引率
3.60%
发文量
98
审稿时长
6-12 weeks
期刊介绍: Advanced molecular research techniques have transformed hematology in recent years. With improved understanding of hematologic diseases, we now have the opportunity to research and evaluate new biological therapies, new drugs and drug combinations, new treatment schedules and novel approaches including stem cell transplantation. We can also expect proteomics, molecular genetics and biomarker research to facilitate new diagnostic approaches and the identification of appropriate therapies. Further advances in our knowledge regarding the formation and function of blood cells and blood-forming tissues should ensue, and it will be a major challenge for hematologists to adopt these new paradigms and develop integrated strategies to define the best possible patient care. Expert Review of Hematology (1747-4086) puts these advances in context and explores how they will translate directly into clinical practice.
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