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血液学中的表观遗传机制和下一代编辑平台:从分子基础到治疗前沿

Minzhi Huang¹, Ahmad Ewadi², Nazli Servatian³

¹College of Professional Studies, Boston Campus, MA 02115, United States.

Critical reviews in oncology/hematology

|September 3, 2025

概括

表观遗传编辑提供了可编程的,可逆的对基因表达的控制,而无需改变DNA. 这项技术正在彻底改变血液病治疗和免疫细胞疗法.

科学领域:

血液学
分子生物学
基因编辑

背景情况:

表观遗传调节对于正常的血细胞发育至关重要,并且在血液病中经常受到调节.
传统的基因编辑方法改变了DNA,但新的表观基因编辑工具可以在不改变DNA序列的情况下逆转基因表达.

研究的目的:

审查表观遗传编辑技术的演变和当前状态.
突出它们在治疗血液恶性瘤和遗传性血液疾病中的应用.
讨论表观遗传编辑在先进细胞治疗中的整合.

主要方法:

探索表观遗传编辑平台,包括指蛋白,TALE和基于CRISPR的系统 (CRISPR-dCas9,dCas12,dCas13).
催化不活的CRISPR变体与染色体或RNA修饰酶的融合.
应用这些工具来精确控制基因表达和表达谱.

主要成果:

表观遗传编辑可以精确调节瘤基因,重新激活瘤抑制剂,并纠正白血病,淋巴瘤,MDS,β- thalassemia和状细胞疾病中的表观遗传错误.
整合到CAR-T和NK细胞疗法中可以提高它们的疗效.
下一代表观遗传编辑为血液疾病提供更安全,更动态,更可调整的干预措施.

结论:

表观遗传编辑代表了精确血液治疗的范式转变.
这些先进的工具为治疗各种血液疾病提供了新的策略.
这一领域正在迅速发展,有望产生重大临床影响.

关键词:

在CRISPR-dCas9中表观遗传编辑血质疾病血造干细胞重编程下一代治疗方法

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