Human iPSC for Therapeutic Approaches to the Nervous System: Present and Future Applications.
Human iPSC for Therapeutic Approaches to the Nervous System: Present and Future Applications.
复制标题
DOI：
10.1155/2016/4869071
复制
发表时间：
2016
影响因子：
4.3
通讯作者：
Germano IM
中科院分区：
文献类型：
--
作者：
Cefalo MG;Carai A;Miele E;Po A;Ferretti E;Mastronuzzi A;Germano IM
包括中风、脊髓损伤（SCI）和脑肿瘤在内的许多中枢神经系统（CNS）疾病是世界范围内发病率/死亡率的重要原因，但还没有令人满意的治疗方法。以细胞为基础的治疗来恢复失去的功能或携带新的治疗基因是一种有前途的新治疗方法，特别是在人类iPSC可用之后。然而，有效产生无足迹和无异种的人iPSC是其临床应用的先决条件。在本文中，我们将首先总结目前获得无足迹和无异种的人iPSC的方法。然后，我们将审查目前的iPSC的应用程序在治疗方法的CNS再生和它们作为载体携带促凋亡基因的脑肿瘤和审查其应用程序的神经系统疾病的建模和制定新的治疗方法。将对现有结果进行总结和比较。最后，我们将讨论目前的限制，阻止iPSC被大规模用于临床应用，并提供了未来的改进领域的概述。总之，在获得适用于神经系统疾病领域临床使用的iPSC方面已经取得了重大进展。目前克服技术挑战的努力，包括减少劳动力和成本，有望加快该技术在临床环境中的整合。
Many central nervous system (CNS) diseases including stroke, spinal cord injury (SCI), and brain tumors are a significant cause of worldwide morbidity/mortality and yet do not have satisfying treatments. Cell-based therapy to restore lost function or to carry new therapeutic genes is a promising new therapeutic approach, particularly after human iPSCs became available. However, efficient generation of footprint-free and xeno-free human iPSC is a prerequisite for their clinical use. In this paper, we will first summarize the current methodology to obtain footprint- and xeno-free human iPSC. We will then review the current iPSC applications in therapeutic approaches for CNS regeneration and their use as vectors to carry proapoptotic genes for brain tumors and review their applications for modelling of neurological diseases and formulating new therapeutic approaches. Available results will be summarized and compared. Finally, we will discuss current limitations precluding iPSC from being used on large scale for clinical applications and provide an overview of future areas of improvement. In conclusion, significant progress has occurred in deriving iPSC suitable for clinical use in the field of neurological diseases. Current efforts to overcome technical challenges, including reducing labour and cost, will hopefully expedite the integration of this technology in the clinical setting.