Induced pluripotent stem cells from ALS patients for disease modeling.
Induced pluripotent stem cells from ALS patients for disease modeling.
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DOI：
10.1016/j.brainres.2014.09.017
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发表时间：
2015-05-14
期刊：
影响因子：
2.9
通讯作者：
Maragakis, Nicholas J.
中科院分区：
文献类型：
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作者：
Richard, Jean-Philippe;Maragakis, Nicholas J.
将成人体细胞重编程为能分化为发育中的人类所有三个胚层的多能干细胞的能力已经从根本上改变了生物医学研究的前景。对于神经退行性疾病，如肌萎缩性侧索硬化症（ALS），这种疾病直到成年后才会表现出来，而且是一种异质性疾病，动物模型很少，这项技术可能特别重要。诱导多能干细胞（iPSC）已经从几种家族性ALS患者以及一些散发性ALS患者中产生。这些细胞已分化为与als相关的细胞亚型，包括运动神经元和星形胶质细胞等。als相关病理也在这些细胞的运动神经元中被发现，这可能为了解体外疾病机制提供了一个窗口。鉴于这是一个相对较新的研究领域，在iPSC方法能够发挥其作为ALS建模工具的潜力以及为ALS治疗方法的研究提供平台之前，仍然存在许多挑战。
The ability to reprogram adult somatic cells into pluripotent stem cells that can differentiate into all three germ layers of the developing human has fundamentally changed the landscape of biomedical research. For a neurodegenerative disease like Amyotrophic Lateral Sclerosis (ALS), which does not manifest itself until adulthood and is a heterogeneous disease with few animal models, this technology may be particularly important. Induced pluripotent stem cells (iPSC) have been created from patients with several familial forms of ALS as well as some sporadic forms of ALS. These cells have been differentiated into ALS-relevant cell subtypes including motor neurons and astrocytes, among others. ALS-relevant pathologies have also been identified in motor neurons from these cells and may provide a window into understanding disease mechanisms in vitro. Given that this is a relatively new field of research, numerous challenges remain before iPSC methodologies can fulfill their potential as tools for modeling ALS as well as providing a platform for the investigation of ALS therapeutics.
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DOI：
10.1016/j.mcn.2013.07.007
发表时间：
2013-09
期刊：
Molecular and cellular neurosciences
影响因子：
--
作者：
Burkhardt MF;Martinez FJ;Wright S;Ramos C;Volfson D;Mason M;Garnes J;Dang V;Lievers J;Shoukat-Mumtaz U;Martinez R;Gai H;Blake R;Vaisberg E;Grskovic M;Johnson C;Irion S;Bright J;Cooper B;Nguyen L;Griswold-Prenner I;Javaherian A
通讯作者：
Javaherian A
影响因子：
3.5
作者：
Mitne-Neto M;Machado-Costa M;Marchetto MC;Bengtson MH;Joazeiro CA;Tsuda H;Bellen HJ;Silva HC;Oliveira AS;Lazar M;Muotri AR;Zatz M
通讯作者：
Zatz M
DOI：
10.1073/pnas.1202922109
发表时间：
2012-04-10
影响因子：
11.1
作者：
Bilican, Bilada;Serio, Andrea;Chandran, Siddharthan
通讯作者：
Chandran, Siddharthan
影响因子：
6
作者：
Haidet-Phillips, Amanda M.;Roybon, Laurent;Maragakis, Nicholas J.
通讯作者：
Maragakis, Nicholas J.
影响因子：
46.9
作者：
通讯作者：
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