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多能干细胞中年轻起病型糖尿病的建模:挑战与成就。

Modeling Maturity Onset Diabetes of the Young in Pluripotent Stem Cells: Challenges and Achievements.

机构信息

The Azrieli Center for Stem Cells and Genetic Research, Department of Genetics, The Alexander Silberman Institute of Life Sciences, The Hebrew University of Jerusalem, Jerusalem, Israel.

出版信息

Front Endocrinol (Lausanne). 2021 Feb 22;12:622940. doi: 10.3389/fendo.2021.622940. eCollection 2021.

Abstract

Maturity onset diabetes of the young (MODY), is a group of monogenic diabetes disorders. Rodent models for MODY do not fully recapitulate the human phenotypes, calling for models generated in human cells. Human pluripotent stem cells (hPSCs), capable of differentiation towards pancreatic cells, possess a great opportunity to model MODY disorders . Here, we review the models for MODY diseases in hPSCs to date and the molecular lessons learnt from them. We also discuss the limitations and challenges that these types of models are still facing.

摘要

青少年发病的成年型糖尿病(MODY)是一组单基因糖尿病疾病。MODY 的啮齿动物模型不能完全重现人类表型,因此需要在人类细胞中生成模型。人类多能干细胞(hPSC)能够向胰腺细胞分化,为模拟 MODY 疾病提供了绝佳机会。本文综述了目前 hPSC 中 MODY 疾病模型及其从中获得的分子知识,并讨论了这些模型仍然面临的局限性和挑战。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b667/7937923/debab88bd063/fendo-12-622940-g001.jpg

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