人类多能干细胞治疗糖尿病肾病的潜力和挑战。

IF 3.3 Q2 BIOCHEMISTRY & MOLECULAR BIOLOGY
Wanyue Xu, Fangyu Yi, Haiyang Liao, Caifeng Zhu, Xiaodi Zou, Yanzhao Dong, Weijie Zhou, Zexing Sun, Jiazhen Yin
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引用次数: 0

摘要

糖尿病肾病(DN)是糖尿病的一种常见并发症,目前的治疗方案效果有限,特别是在晚期。人多能干细胞(hPSCs),特别是诱导的多能干细胞(iPSCs),由于其多能性、向肾脏特异性细胞分化的能力和个性化治疗的适用性,在DN的治疗中显示出巨大的潜力。基于ipsc的个性化方法可以有效减轻免疫排斥反应,这是同种异体移植的常见挑战,从而提高治疗效果。聚集规律间隔短回文重复序列(CRISPR)基因编辑通过精确校正疾病相关遗传缺陷,提高治疗细胞的安全性和有效性,进一步增强了人造血干细胞的潜力。除了直接治疗外,hPSCs在疾病建模和药物筛选方面也被证明是有价值的,特别是在识别和验证疾病特异性靶点方面。来源于人造血干细胞的肾类器官复制了DN病理的关键特征,使其成为验证治疗靶点和评估药物疗效的有用平台。相比之下,在临床前模型中,hPSCs和间充质SCs (MSCs)都显示出改善肾功能的希望,其中hPSCs具有更广泛的分化能力。结合组织工程技术,如三维生物打印和生物工程支架,通过支持功能性肾脏结构的形成和增强体内整合和再生能力,扩大了hPSCs的再生潜力。尽管目前面临着诸如致瘤性、基因组不稳定性和有限的直接研究等挑战,但基因编辑、分化方案和组织工程方面的进展有望解决这些障碍。这些方法的持续优化可能会导致造血干细胞成功的临床应用,可能会彻底改变DN的治疗选择。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The Potential and Challenges of Human Pluripotent Stem Cells in the Treatment of Diabetic Nephropathy.

Diabetic nephropathy (DN) is a prevalent complication of diabetes, with current treatment options offering limited effectiveness, particularly in advanced stages. Human pluripotent stem cells (hPSCs), particularly induced PSCs (iPSCs), show promising potential in the treatment of DN due to their pluripotency, capacity for differentiation into kidney-specific cells, and suitability for personalized therapies. iPSC-based personalized approaches can effectively mitigate immune rejection, a common challenge with allogeneic transplants, thus enhancing therapeutic outcomes. Clustered regularly interspaced short palindromic repeats (CRISPR) gene editing further enhances the potential of hPSCs by enabling the precise correction of disease-associated genetic defects, increasing both the safety and efficacy of therapeutic cells. In addition to direct treatment, hPSCs have proven valuable in disease modeling and drug screening, particularly for identifying and validating disease-specific targets. Kidney organoids derived from hPSCs replicate key features of DN pathology, making them useful platforms for validating therapeutic targets and assessing drug efficacy. Comparatively, both hPSCs and mesenchymal SCs (MSCs) have shown promise in improving renal function in preclinical models, with hPSCs offering broader differentiation capacity. Integration with tissue engineering technologies, such as three-dimensional bioprinting and bioengineered scaffolds, expands the regenerative potential of hPSCs by supporting the formation of functional renal structures and enhancing in vivo integration and regenerative capacity. Despite current challenges, such as tumorigenicity, genomic instability, and limited direct research, advances in gene editing, differentiation protocols, and tissue engineering promise to address these barriers. Continued optimization of these approaches will likely lead to successful clinical applications of hPSCs, potentially revolutionizing treatment options for DN.

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