相关实验视频

Updated: May 7, 2025

Conversion of Human Induced Pluripotent Stem Cells iPSCs into Functional Spinal and Cranial Motor Neurons Using PiggyBac Vectors

Conversion of Human Induced Pluripotent Stem Cells iPSCs into Functional Spinal and Cranial Motor Neurons Using PiggyBac Vectors

Published on: May 1, 2019

人类iPSC衍生的神经干细胞植入并改善MPS I小鼠的病理生理学

Caitlin C Calhoun¹, Shih-Hsin Kan¹, Alexander E Stover¹

¹Research Institute, Children's Hospital of Orange County, Orange, CA, USA.

Molecular therapy. Methods & clinical development

|January 7, 2025

概括

诱导的神经干细胞 (hiNSCs) 提供了一种有前途的细胞疗法,用于I型粘多糖症 (MPS I). 这些细胞在小鼠模型中显示出迁移和纠正酶缺乏,这表明治疗这种罕见的代谢障碍的潜力.

科学领域:

干细胞疗法是一种干细胞疗法.
神经科学是一个神经科学.
代谢障碍代谢障碍代谢障碍

背景情况:

第一种类型的粘多糖症 (MPS I) 是由α-L-氨基酶 (IDUA) 缺乏引起的遗传性疾病,导致有毒的葡萄糖氨基酸糖的积累.
目前的治疗方法,如酶替代疗法 (ERT) 和造血干细胞移植 (HSCT),在解决因血脑屏障 (BBB) 挑战而导致的神经退行症方面有效性有限.

研究的目的:

研究人类诱导的神经干细胞 (hiNSCs) 作为MPS I的基于细胞治疗的潜力.
评估hiNSCs迁移的能力,纠正IDUA缺乏,并在MPS I小鼠模型的中枢神经系统 (CNS) 中植入.

主要方法:

将人类带血细胞重新编程成诱导多能干细胞 (iPSC),然后分化为hiNSC.
在新生儿免疫缺陷MPS I小鼠中移植hiNSCs.
对IDUA活性,hiNSC分布,分化为质细胞以及疾病生物标志物的变化进行大脑组织分析.

主要成果:

在体外研究证实了hiNSC迁移和IDUA缺乏的交叉纠正.
在MPS I小鼠的移植后分析显示,IDUA活动在大脑中的部分恢复.
植入的hiNSCs分化为质细胞,并在整个大脑中分布,与关键疾病生物标志物的水平降低有关 (例如β-hexosaminidase,CD68,LAMP1).

结论:

人类诱导的神经干细胞 (hiNSCs) 显示出作为MPS I患者特异性细胞治疗的潜力.
hiNSC移植可能提供一种新的策略,通过向中枢神经系统病理来克服当前MPS I治疗的局限性.

关键词:

在CNS中,CNS是CNS.CXCR4CXCR4CXCR4CXCR4CXCR4CXCR4CXCR4CXCR4CXCR4CXCR4C 国家安全委员会 (NSC) 的国家安全委员会.细胞分化的细胞分化.基于细胞和组织的疗法.这是一种遗传性遗传.诱导多能干细胞诱导干细胞.这是一种炎症炎症炎症炎症.神经干细胞的神经干细胞神经退行性疾病的神经退行性疾病

更多相关视频

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

Derivation of Adult Human Fibroblasts and their Direct Conversion into Expandable Neural Progenitor Cells

Derivation of Adult Human Fibroblasts and their Direct Conversion into Expandable Neural Progenitor Cells

Published on: July 29, 2015

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 types of cells. Ordinarily, cells that have differentiated into a specific cell type are post-mitotic—that is, they no longer divide. However, scientists have found a way to reprogram these mature cells so that they “de-differentiate” and return to an unspecialized, proliferative state. These cells are also pluripotent like embryonic stem cells—able to produce all cell types—and are therefore...