Advances in cell models for inherited thrombocytopenia
10.13303/j.cjbt.issn.1004-549x.2025.12.023
- VernacularTitle:遗传性血小板减少症：细胞模型研究进展
- Author:
Yangzhou JIANG
1
;
Chengning TAN
1
;
Zhongjun LI
1
;
Li CHEN
1
Author Information
1. Department of Transfusion Medicine, The Second Affiliated Hospital, Army Medical University, Chongqing 400037, China
- Publication Type:Journal Article
- Keywords:
inherited thrombocytopenia;
cell models;
immortalized stem cell lines;
immortalized megakaryocyte cell lines
- From:
Chinese Journal of Blood Transfusion
2025;38(12):1801-1811
- CountryChina
- Language:Chinese
-
Abstract:
Inherited thrombocytopenia (IT) is a group of rare hereditary platelet disorders characterized by reduced platelet counts and, sometimes accompanied by platelet dysfunction. This review comprehensively summarizes various cell models used in IT research, which are derived from different sources, including patient-derived primary cells (such as megakaryocytes, platelets, and hematopoietic stem and progenitor cells), stem cell lines (encompassing both adult and pluripotent stem cells), and immortalized megakaryocyte cell lines derived from tumor cells. These cell models provide an indispensable research platform for in-depth exploration of the pathogenesis of IT, screening of potential therapeutic drugs, and evaluation of drug efficacy. Additionally, this review delves into the research progress of utilizing gene editing technologies to simulate IT-causing mutations and their pathogenic mechanisms, and summarizes the application prospects and challenges of in vitro cell models in the field of IT research.
