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用于疾病建模的干细胞类器官基因组工程

Genome engineering of stem cell organoids for disease modeling.

作者信息

Sun Yingmin, Ding Qiurong

机构信息

CAS Key Laboratory of Nutrition and Metabolism, Institute for Nutritional Sciences, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai, 200031, China.

University of Chinese Academy of Sciences, Shanghai, 200031, China.

出版信息

Protein Cell. 2017 May;8(5):315-327. doi: 10.1007/s13238-016-0368-0. Epub 2017 Jan 19.

DOI:10.1007/s13238-016-0368-0
PMID:28102490
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5413597/
Abstract

Precision medicine emerges as a new approach that takes into account individual variability. Successful realization of precision medicine requires disease models that are able to incorporate personalized disease information and recapitulate disease development processes at the molecular, cellular and organ levels. With recent development in stem cell field, a variety of tissue organoids can be derived from patient specific pluripotent stem cells and adult stem cells. In combination with the state-of-the-art genome editing tools, organoids can be further engineered to mimic disease-relevant genetic and epigenetic status of a patient. This has therefore enabled a rapid expansion of sophisticated in vitro disease models, offering a unique system for fundamental and biomedical research as well as the development of personalized medicine. Here we summarize some of the latest advances and future perspectives in engineering stem cell organoids for human disease modeling.

摘要

精准医学作为一种考虑个体差异的新方法应运而生。成功实现精准医学需要能够整合个性化疾病信息并在分子、细胞和器官水平上重现疾病发展过程的疾病模型。随着干细胞领域的最新发展,可以从患者特异性多能干细胞和成体干细胞中获得多种组织类器官。结合最先进的基因组编辑工具,类器官可以进一步工程化,以模拟患者与疾病相关的遗传和表观遗传状态。因此,这使得复杂的体外疾病模型迅速扩展,为基础研究和生物医学研究以及个性化医学的发展提供了一个独特的系统。在这里,我们总结了用于人类疾病建模的工程化干细胞类器官的一些最新进展和未来前景。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/06d1/5413597/40050bb62564/13238_2016_368_Fig2_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/06d1/5413597/669b567d6b00/13238_2016_368_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/06d1/5413597/40050bb62564/13238_2016_368_Fig2_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/06d1/5413597/669b567d6b00/13238_2016_368_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/06d1/5413597/40050bb62564/13238_2016_368_Fig2_HTML.jpg

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Genome engineering of stem cell organoids for disease modeling.用于疾病建模的干细胞类器官基因组工程
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Stem cells for organoids.用于类器官的干细胞。
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Human retinal organoids with an OPA1 mutation are defective in retinal ganglion cell differentiation and function.携带有 OPA1 突变的人类视网膜类器官在视网膜神经节细胞分化和功能上存在缺陷。
Stem Cell Reports. 2024 Jan 9;19(1):68-83. doi: 10.1016/j.stemcr.2023.11.004. Epub 2023 Dec 14.
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Engineered organoids in oral and maxillofacial regeneration.口腔颌面再生中的工程化类器官

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