人类iPSC技术的临床翻译:进展,安全问题和未来方向
Sarah Dhaiban1, Sanjana Chandran2, Mohammed Noshi3
1Department of Biological Sciences, Khalifa University of Science and Technology, Abu Dhabi, United Arab Emirates.
Frontiers in cell and developmental biology
|October 8, 2025
概括
人类诱导的多能干细胞 (hiPSCs) 提供个性化的再生医学,但面临临临床翻译挑战. 基因编辑和人工智能的进步正在改善安全性和制造,使hiPSC疗法更接近广泛使用.
科学领域:
- 再生医学是一种再生医学.
- 干细胞生物学 干细胞生物学
- 生物技术是生物技术.
背景情况:
- 人类诱导多能干细胞 (hiPSC) 是从成年细胞中衍生出来的,为再生疗法提供患者特异性的细胞.
- hiPSC对疾病建模,药物测试和具有减少免疫排斥的个性化治疗具有前景.
- 由于分化变异性,免疫反应,遗传不稳定性和瘤风险,hiPSCs的临床转化受到阻碍.
研究的目的:
- 审查目前转化hiPSC技术用于临床应用的进展和挑战.
- 评估安全性,制造和监管障碍,影响基于hiPSC的疗法的广泛采用.
- 讨论新兴技术,可以加速hiPSCs的临床整合.
主要方法:
- 关于hiPSC技术和临床翻译的最新科学文献的综述.
- 对正在进行的临床试验及其报告结果的分析.
- 评估技术进步,包括基因编辑和干细胞分化中的AI.
主要成果:
- 目前正在进行的视网膜,神经和心脏疾病等领域的临床试验表明hiPSC疗法的潜在可行性和安全性.
- 克里斯普尔/Cas9基因编辑和人工智能引导的差异化正在提高iPSC质量和针对患者的治疗量身定制.
- 在良好生产规范条件下的大规模制造,长期植入和免疫耐受性方面仍然存在重大挑战.
结论:
- hiPSC技术已经取得了显著的进步,早期临床试验结果充满希望.
- 克服制造,安全和监管方面的挑战对于广泛的临床采用至关重要.
- 新兴技术提供了提高hiPSC质量的途径,并加速将其整合到常规医疗实践中.
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