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类器官在儿童遗传性疾病中的应用研究进展

Research progress on the application of organoids in genetic diseases of childhood
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摘要 类器官是高度保留其来源组织器官特性的体外3D组织培养物,在生物医学领域得到了广泛的应用。类器官可以作为研究疾病的体外模型,有助于进一步了解疾病的病理生理和分子机制,为遗传性疾病的个性化治疗提供了技术支撑。该文将围绕类器官技术在儿童遗传性疾病中的应用,着重介绍其在模拟疾病模型、矫正遗传缺陷、个性化治疗方面的优势及潜在的发展前景,以期为后续研究提供参考。 Organoids are in vitro 3D tissue cultures that retain the characteristics of their source tissue and organs,and have been widely used in biomedical fields.Organoids have emerged as effective models for investigating diseases,contribute to further understanding of pathophysiology and molecular mechanisms of diseases,and provide technical support for personalized treatment of genetic diseases.This article specifically explores the application of organoid technology in childhood genetic diseases.It emphasizes the advantages and potential advancements of this technology in disease modeling,correction of genetic defects and personalized treatment,thereby serving as a valuable reference for future research endeavors.
作者 雷敏 蔡春泉 LEI Min;CAI Chunquan(Tianjin Children's Hospital/Children's Hospital of Tianjin University,Tianjin 300134,China;Medical School of Tianjin University;Tianjin Institute of Pediatrics;Tianjin Key Laboratory of Birth Defects for Prevention and Treatment△Corresponding Author E-mail:cqcns6@126.com)
出处 《天津医药》 CAS 2024年第1期33-37,共5页 Tianjin Medical Journal
基金 天津市卫生健康委员会科技项目(TJWJ2021ZD007) 天津市科技计划项目(21JCZDJC00390)。
关键词 类器官 芯片分析技术 遗传性疾病 先天性 基因编辑 药物评价 临床前 药物筛选 organoids microchip analytical procedures genetic diseases,inborn gene diting drug evaluation,preclinical drug screening assays
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