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发明公开
CN109679922A CADASIL病人特异性血管平滑肌细胞与血管内皮细胞的制备方法 审中-实审

CADASIL病人特异性血管平滑肌细胞与血管内皮细胞的制备方法
摘要：
本发明公开了CADASIL病人特异性血管平滑肌细胞与内皮细胞的制备方法。本发明提供了一种制备CADASIL疾病细胞模型的方法，包括如下步骤：a)将来源于携带NOTCH3基因杂合突变的CADASIL病人的离体成纤维细胞进行重编程得到诱导多能干细胞系；b)将所述诱导多能干细胞系定向分化为血管壁细胞，得到CADASIL疾病细胞模型。本发明将利用重编程技术和定向分化技术在体外获得CADASIL患者特异的诱导多能干细胞，并将其定向分化成血管平滑肌细胞(VSMC)与血管内皮细胞(VEC)，用于研究CADASIL小动脉病变的发病机制，并为药物筛选提供平台。
摘要（英）：

The invention discloses a method for preparing specific vascular smooth muscle cells and vascular endothelial cells of CADASIL patients. The method for preparing a CADASIL disease cell model providedby the invention comprises the following steps: a) reprogramming in vitro fibroblasts from a CADASIL patient carrying NOTCH3 gene heterozygous mutation, thereby acquiring an induced pluripotent stem cell line; b) directionally differentiating the induced pluripotent stem cell line into vascular wall cells, thereby acquiring the CADASIL disease cell model. According to the invention, a reprogramming technique and a directional differentiation technique are utilized to in vitro acquire the specific induced pluripotent stem cells of CADASIL patients, the specific induced pluripotent stem cells are directionally differentiated into vascular smooth muscle cells (VSMC) and vascular endothelial cells (VEC) which are used for researching pathogenesis of CADASIL arteriole lesion, and a platform issupplied for drug screening.

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