从两名系统性硬化症患者的脂肪来源间充质基质细胞中生成人诱导多能干细胞系（iPSC）。

Generation of human induced pluripotent stem cell lines (iPSC) from adipose-derived mesenchymal stromal cells from two patients with systemic sclerosis.

作者信息

Velier Mélanie, Appay Romain, Arcani Robin, Desprat Romain, Simoncini Stéphanie, Zavarro Anouck, Noel Danièle, Bendaass Lenha, Jorgensen Christian, Gomes De Pinho Quentin, Benyamine Audrey, Granel Brigitte, Daumas Aurélie, Dignat George Francoise, Sabatier Florence, Magalon Jérémy

机构信息

Aix-Marseille Univ, C2VN, INSERM 1263, INRAE1260, Marseille, France; Culture and Cell Therapy Laboratory, INSERM CIC BT 1409, Hôpital de la Conception, Assistance Publique Hôpitaux de Marseille (AP-HM), Marseille, France.

Department of Pathological Anatomy and Neuropathology, Hôpital de La Timone, Public Assistance Publique Hôpitaux de Marseille (AP-HM), Marseille, France; Aix-Marseille Univ, CNRS, Institute of Neurophysiopathology, Marseille, France.

出版信息

Stem Cell Res. 2025 Feb;82:103624. doi: 10.1016/j.scr.2024.103624. Epub 2024 Dec 5.

DOI:10.1016/j.scr.2024.103624

PMID:39647347

Abstract

Systemic sclerosis (SSc) is a rare and complex connective tissue disease associated with high morbidity and mortality. SSc is characterized by ischemic vasculopathy, cutaneous and visceral fibrosis and a dysimmune state (Denton and Khanna, 2017; Volkmann et al., 2023; Barnes and Mayes, 2012). We have derived induced pluripotent stem cell (iPSC) lines from two SSc patients aged 38 and 67 years with severe vascular phenotype. These iPSC lines expressed pluripotent markers, exhibited normal and stable genome, and differentiated into trilineage embryonic layers in teratoma formation assays. These SSc-specific iPSC lines can be differentiated into endothelial cells, providing a valuable model to elucidate vascular dysfunction and develop personalized therapeutic approaches.

摘要

系统性硬化症（SSc）是一种罕见且复杂的结缔组织疾病，其发病率和死亡率都很高。SSc的特征是缺血性血管病变、皮肤和内脏纤维化以及免疫失调状态（丹顿和坎纳，2017年；福尔克曼等人，2023年；巴恩斯和梅斯，2012年）。我们从两名患有严重血管表型的38岁和67岁SSc患者中获得了诱导多能干细胞（iPSC）系。这些iPSC系表达多能性标志物，具有正常且稳定的基因组，并在畸胎瘤形成试验中分化为三胚层胚胎层。这些SSc特异性iPSC系可分化为内皮细胞，为阐明血管功能障碍和开发个性化治疗方法提供了有价值的模型。