相关实验视频

Updated: Jul 17, 2025

Generation of iPSC-derived Human Brain Organoids to Model Early Neurodevelopmental Disorders

Generation of iPSC-derived Human Brain Organoids to Model Early Neurodevelopmental Disorders

Published on: April 14, 2017

人类iPSC作为BMP相关罕见疾病的模型系统

Gonzalo Sánchez-Duffhues^1,2, Christian Hiepen³

¹Nanomaterials and Nanotechnology Research Center (CINN-CSIC), ISPA-HUCA, Avda. de Roma, s/n, 33011 Oviedo, Spain.

|September 8, 2023

概括

基于诱导多能干细胞 (iPSC) 的模型为与骨形态遗传蛋白 (BMP) 信号相关的罕见遗传疾病提供了改进的药物开发. 对于这些复杂的人性化系统来说,仔细的协议优化至关重要.

科学领域:

生物化学生物化学
遗传学遗传学是一个
干细胞生物学干细胞生物学

背景情况:

骨形态遗传蛋白 (BMP) 信号通路障碍与罕见的遗传性疾病有关,如纤维化成形性骨渐进性 (FOP),肺动脉高血压 (PAH) 和遗传性出血性透视 (HHT).
几十年的动物研究已经建立了对BMP信号传递分子机制的基础知识.
诱导多能干细胞 (iPSCs) 为研究疾病和药物开发提供了一个人性化的体外环境.

研究的目的:

审查目前关于iPSC衍生模型系统的文献,以研究与BMP信号相关的罕见疾病.
突出与患者衍生的iPSC模型相关的挑战,包括源材料访问和潜在的并发症.
讨论复杂的体外模型的进展,以模拟组织微环境.

主要方法:

在BMP信号研究中对基于iPSC的模型的文献综述.
分析产生和区分IPSC与BMP途径突变患者的挑战.
探索新兴复杂的体外培养技术 (器官,芯片上的器官,微流体).

主要成果:

来自患者的iPSC模型在人性化的背景下有望改善药物疗效,特异性和毒性评估.
在BMP信号传输中获得或失去功能的突变可以阻碍iPSC生成,维护和差异化,需要协议优化.
现在正在出现包括细胞力学,细胞外矩阵 (ECM) 和有机体/器官在芯片技术的先进体外模型.

结论:

基于iPSC的模型代表了在罕见的BMP信号障碍中药物开发的重大进展.
优化协议对于克服与使用患者衍生的iPSCs相关的挑战至关重要.
未来的研究方向包括开发复杂的体外模型,重现复杂的组织微环境.

关键词:

在 FAP FAP 里面.在 FOP FOP 里面.HHT HHT HHT 的时间.这些PAH是PAH.这就是TGF-β.

更多相关视频

Generation of 3D Skin Organoid from Cord Blood-derived Induced Pluripotent Stem Cells

Generation of 3D Skin Organoid from Cord Blood-derived Induced Pluripotent Stem Cells

Published on: April 18, 2019

Rapid Isolation of BMPR-IB+ Adipose-Derived Stromal Cells for Use in a Calvarial Defect Healing Model

Rapid Isolation of BMPR-IB+ Adipose-Derived Stromal Cells for Use in a Calvarial Defect Healing Model

Published on: February 24, 2017

相关概念视频

EPS and iPS Cells in Disease Research

EPS and iPS Cells in Disease Research

Embryonic and induced pluripotent stem cells are excellent models for disease research because of their ability to self-renew and differentiate into most cell types. Somatic cells from a patient are isolated and reprogrammed into induced pluripotent stem cells or iPSCs. These iPSCs are later differentiated into the desired cell type, which mirrors the diseased cell of the patient. In this way, disease models have been created for investigating diseases such as Down syndrome, type I diabetes,...

iPS Cell Differentiation

iPS Cell Differentiation

The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.

Induced Pluripotent Stem Cells

Induced Pluripotent Stem Cells

Stem cells are undifferentiated cells that divide and produce different cell types. Ordinarily, cells that have differentiated into a specific cell type are terminally differentiated; however, scientists have found a way to reprogram these mature cells so that they dedifferentiate and return to an unspecialized, proliferative state. These cells are pluripotent like embryonic stem cells—able to produce all cell types—and are called induced pluripotent stem cells (iPSCs).
Somatic...