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Induced pluripotent stem cells: applications in regenerative medicine disease modeling and drug discovery

机译:诱导性多能干细胞:在再生医学疾病建模和药物发现中的应用

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摘要

Recent progresses in the field of Induced Pluripotent Stem Cells (iPSCs) have opened up many gateways for the research in therapeutics. iPSCs are the cells which are reprogrammed from somatic cells using different transcription factors. iPSCs possess unique properties of self renewal and differentiation to many types of cell lineage. Hence could replace the use of embryonic stem cells (ESC), and may overcome the various ethical issues regarding the use of embryos in research and clinics. Overwhelming responses prompted worldwide by a large number of researchers about the use of iPSCs evoked a large number of peple to establish more authentic methods for iPSC generation. This would require understanding the underlying mechanism in a detailed manner. There have been a large number of reports showing potential role of different molecules as putative regulators of iPSC generating methods. The molecular mechanisms that play role in reprogramming to generate iPSCs from different types of somatic cell sources involves a plethora of molecules including miRNAs, DNA modifying agents (viz. DNA methyl transferases), NANOG, etc. While promising a number of important roles in various clinical/research studies, iPSCs could also be of great use in studying molecular mechanism of many diseases. There are various diseases that have been modeled by uing iPSCs for better understanding of their etiology which maybe further utilized for developing putative treatments for these diseases. In addition, iPSCs are used for the production of patient-specific cells which can be transplanted to the site of injury or the site of tissue degeneration due to various disease conditions. The use of iPSCs may eliminate the chances of immune rejection as patient specific cells may be used for transplantation in various engraftment processes. Moreover, iPSC technology has been employed in various diseases for disease modeling and gene therapy. The technique offers benefits over other similar techniques such as animal models. Many toxic compounds (different chemical compounds, pharmaceutical drugs, other hazardous chemicals, or environmental conditions) which are encountered by humans and newly designed drugs may be evaluated for toxicity and effects by using iPSCs. Thus, the applications of iPSCs in regenerative medicine, disease modeling, and drug discovery are enormous and should be explored in a more comprehensive manner.
机译:诱导性多能干细胞(iPSC)领域的最新进展为治疗学的研究打开了许多门户。 iPSC是使用不同转录因子从体细胞重新编程的细胞。 iPSC具有自我更新和分化为多种类型细胞谱系的独特特性。因此可以替代胚胎干细胞(ESC)的使用,并且可以克服有关研究和临床中使用胚胎的各种伦理问题。在全球范围内,大量研究人员对iPSC的使用产生了压倒性的反应,这引起了许多人对iPSC生成建立更可靠方法的认识。这将需要详细了解底层机制。有大量报告显示不同分子作为iPSC生成方法的假定调节剂的潜在作用。在重编程以从不同类型的体细胞来源生成iPSC中发挥作用的分子机制涉及许多分子,包括miRNA,DNA修饰剂(即DNA甲基转移酶),NANOG等。在临床/研究中,iPSCs在研究许多疾病的分子机制方面也可能具有很大的用途。通过使用iPSC可以对各种疾病进行建模,以更好地了解其病因,并可以将其进一步用于开发针对这些疾病的推定治疗方法。另外,iPSC被用于产生患者特异性细胞,其可以被移植到由于各种疾病状况导致的损伤部位或组织变性部位。 iPSC的使用可以消除免疫排斥的机会,因为患者特异性细胞可以用于各种植入过程中的移植。此外,iPSC技术已被用于各种疾病的疾病建模和基因治疗。该技术提供了优于其他类似技术(例如动物模型)的优势。人体和新设计的药物会遇到许多有毒化合物(不同的化学化合物,药物,其他危险化学品或环境条件),可以使用iPSC评估其毒性和作用。因此,iPSC在再生医学,疾病建模和药物发现中的应用是巨大的,应该以更全面的方式进行探索。

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