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Stem cell-based therapy for human diseases
Signal Transduction and Targeted Therapy ( IF 40.8 ) Pub Date : 2022-08-06 , DOI: 10.1038/s41392-022-01134-4
Duc M Hoang 1 , Phuong T Pham 2 , Trung Q Bach 1 , Anh T L Ngo 2 , Quyen T Nguyen 1 , Trang T K Phan 1 , Giang H Nguyen 1 , Phuong T T Le 1 , Van T Hoang 1 , Nicholas R Forsyth 3 , Michael Heke 4 , Liem Thanh Nguyen 1
Affiliation  

Recent advancements in stem cell technology open a new door for patients suffering from diseases and disorders that have yet to be treated. Stem cell-based therapy, including human pluripotent stem cells (hPSCs) and multipotent mesenchymal stem cells (MSCs), has recently emerged as a key player in regenerative medicine. hPSCs are defined as self-renewable cell types conferring the ability to differentiate into various cellular phenotypes of the human body, including three germ layers. MSCs are multipotent progenitor cells possessing self-renewal ability (limited in vitro) and differentiation potential into mesenchymal lineages, according to the International Society for Cell and Gene Therapy (ISCT). This review provides an update on recent clinical applications using either hPSCs or MSCs derived from bone marrow (BM), adipose tissue (AT), or the umbilical cord (UC) for the treatment of human diseases, including neurological disorders, pulmonary dysfunctions, metabolic/endocrine-related diseases, reproductive disorders, skin burns, and cardiovascular conditions. Moreover, we discuss our own clinical trial experiences on targeted therapies using MSCs in a clinical setting, and we propose and discuss the MSC tissue origin concept and how MSC origin may contribute to the role of MSCs in downstream applications, with the ultimate objective of facilitating translational research in regenerative medicine into clinical applications. The mechanisms discussed here support the proposed hypothesis that BM-MSCs are potentially good candidates for brain and spinal cord injury treatment, AT-MSCs are potentially good candidates for reproductive disorder treatment and skin regeneration, and UC-MSCs are potentially good candidates for pulmonary disease and acute respiratory distress syndrome treatment.



中文翻译:


基于干细胞的人类疾病疗法



干细胞技术的最新进展为患有尚未治疗的疾病和病症的患者打开了一扇新的大门。基于干细胞的疗法,包括人类多能干细胞(hPSC)和多能间充质干细胞(MSC),最近已成为再生医学的关键参与者。 hPSC 被定义为自我更新的细胞类型,具有分化为人体各种细胞表型的能力,包括三个胚层。根据国际细胞与基因治疗学会 (ISCT) 的说法,间充质干细胞是多能祖细胞,具有自我更新能力(体外有限)和分化为间充质谱系的潜力。本综述提供了最近使用源自骨髓 (BM)、脂肪组织 (AT) 或脐带 (UC) 的 hPSC 或 MSC 治疗人类疾病的临床应用的最新信息,包括神经系统疾病、肺功能障碍、代谢性疾病等。 /内分泌相关疾病、生殖障碍、皮肤烧伤和心血管疾病。此外,我们讨论了我们自己在临床环境中使用 MSC 进行靶向治疗的临床试验经验,我们提出并讨论了 MSC 组织起源概念以及 MSC 起源如何促进 MSC 在下游应用中的作用,最终目标是促进再生医学转化研究到临床应用。 这里讨论的机制支持所提出的假设,即 BM-MSC 是脑和脊髓损伤治疗的潜在良好候选者,AT-MSC 是生殖障碍治疗和皮肤再生的潜在良好候选者,而 UC-MSC 是肺部疾病的潜在良好候选者和急性呼吸窘迫综合征的治疗。

更新日期:2022-08-06
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