Cellular scRNA-seq of human umbilical cord mesenchymal stem cell (HUCMSC) and human umbilical vein endothelial cells (HUVEC)

We report the first application of human umbilical cord mesenchymal stem cell (HUCMSC)-derived therapy for pulmonary arterial hypertension (PAH). A 3-year-old female presented with heritable PAH (ACVLR1 missense mutation) associated with hereditary hemorrhagic telangiectasia (HHT). Serial intra-pulmonary arterial and centrally intravenous infusions of conditioned media (CM) from allogenic HUCMSCs resulted in marked clinical and hemodynamic improvement after six months. A consecutive multiple cord single-cell RNA-seq integrated analysis (HUCMSC and HUVEC control) revealed eight common cell clusters, of which two (overrepresented by HUCMSCs) had regenerative potential. scRNA profiles of HUCMSC and HUVEC.

本研究首次报道了人脐带间充质干细胞（human umbilical cord mesenchymal stem cell, HUCMSC）来源的疗法用于肺动脉高压（pulmonary arterial hypertension, PAH）的临床应用。一例3岁女性患者罹患携带ACVLR1错义突变、伴有遗传性出血性毛细血管扩张症（hereditary hemorrhagic telangiectasia, HHT）的遗传性肺动脉高压。对其连续经肺动脉及中心静脉输注同种异体人脐带间充质干细胞条件培养基（conditioned media, CM）后，患者在6个月后临床症状及血流动力学指标均获得显著改善。通过对多份脐带样本开展连续单细胞RNA测序整合分析（包含人脐带间充质干细胞及人脐静脉内皮细胞（human umbilical vein endothelial cell, HUVEC）对照），共鉴定出8个共有细胞簇，其中2个在人脐带间充质干细胞中富集并具备再生潜能。本研究同时提供了人脐带间充质干细胞与人脐静脉内皮细胞的单细胞RNA测序图谱。