Derivation of autism spectrum disorder-specific induced pluripotent stem cells from peripheral blood mononuclear cells.

Derivation of autism spectrum disorder-specific induced pluripotent stem cells from peripheral blood mononuclear cells.
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DOI:
10.1016/j.neulet.2012.02.086
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发表时间:
2012-05-10
影响因子:
2.5
通讯作者:
Dykxhoorn DM
Dykxhoorn DM
中科院分区:
医学4区
文献类型:
--
作者:
DeRosa BA;Van Baaren JM;Dubey GK;Lee JM;Cuccaro ML;Vance JM;Pericak-Vance MA;Dykxhoorn DM

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诱导多能干细胞(IPSCs)作为一种生物工具,通过建立相关的细胞模型来揭示疾病的病理生理学,以及作为干细胞的来源用于基于细胞的治疗应用,具有巨大的潜力。通常,IPSCs是通过转基因过表达与祖细胞或干细胞功能相关的转录因子在来自皮肤活检的成纤维细胞中获得的。然而,需要皮肤穿孔活检来获得重新编程的成纤维细胞,这可能会阻碍某些个体群体的参与研究,包括患有自闭症谱系障碍(ASD)的儿童。此外,在非临床环境下获取皮肤穿孔活检是一项挑战。避免这些限制的一个潜在机制是使用外周血单个核细胞(PBMCs)作为重新编程的细胞来源。在这篇文章中,我们描述了从自闭症儿童全血中分离的PBMC的IPSC系的来源,以及它们随后在GABA能神经元中的分化。
Induced pluripotent stem cells (iPSCs) hold tremendous potential both as a biological tool to uncover the pathophysiology of disease by creating relevant cell models and as a source of stem cells for cell-based therapeutic applications. Typically, iPSCs have been derived by the transgenic overexpression of transcription factors associated with progenitor cell or stem cell function in fibroblasts derived from skin biopsies. However, the need for skin punch biopsies to derive fibroblasts for reprogramming can present a barrier to study participation among certain populations of individuals, including children with autism spectrum disorders (ASDs). In addition, the acquisition of skin punch biopsies in non-clinic settings presents a challenge. One potential mechanism to avoid these limitations would be the use of peripheral blood mononuclear cells (PBMCs) as the source of the cells for reprogramming. In this article we describe the derivation of iPSC lines from PBMCs isolated from the whole blood of autistic children, and their subsequent differentiation in GABAergic neurons.
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