类器官在儿童遗传性疾病中的应用研究进展

doi:10.11958/20231509

天津医药 ›› 2024, Vol. 52 ›› Issue (1): 33-37.doi: 10.11958/20231509

• 专题研究·类器官与器官芯片（主编·陈怀永） • 上一篇下一篇

类器官在儿童遗传性疾病中的应用研究进展

雷敏¹^,²^,³(), 蔡春泉¹^,³^,⁴^,^△()

1.天津市儿童医院/天津大学儿童医院（邮编300134）
2.天津大学医学院
3.天津市儿科研究所
4.天津市儿童缺陷防治重点实验室

收稿日期:2023-11-13 出版日期:2024-01-15 发布日期:2024-01-18
通讯作者: ^△E-mail：cqcns6@126.com
作者简介:雷敏（2000），女，硕士在读，主要从事儿科学方面研究。E-mail：2398993952@qq.com
基金资助:
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)

Research progress on the application of organoids in genetic diseases of childhood

LEI Min¹^,²^,³(), CAI Chunquan¹^,³^,⁴^,^△()

1. Tianjin Children's Hospital/Children’s Hospital of Tianjin University, Tianjin 300134, China
2. Medical School of Tianjin University
3. Tianjin Institute of Pediatrics
4. Tianjin Key Laboratory of Birth Defects for Prevention and Treatment

Received:2023-11-13 Published:2024-01-15 Online:2024-01-18
Contact: ^△E-mail：cqcns6@126.com

雷敏, 蔡春泉. 类器官在儿童遗传性疾病中的应用研究进展[J]. 天津医药, 2024, 52(1): 33-37.

LEI Min, CAI Chunquan. Research progress on the application of organoids in genetic diseases of childhood[J]. Tianjin Medical Journal, 2024, 52(1): 33-37.

摘要/Abstract

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。

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

Abstract:

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.

Key words: organoids, microchip analytical procedures, genetic diseases, inborn, gene diting, drug evaluation, preclinical, drug screening assays

中图分类号:

R725

参考文献 36

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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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