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人类诱导多能干细胞:从基础研究到癌症的潜在临床应用。

Human induced pluripotent stem cells from basic research to potential clinical applications in cancer.

机构信息

National Cancer Institute (INCA), Bone Marrow Transplantation Center (CEMO), Laboratory Division, 20230-130 Rio de Janeiro, RJ, Brazil.

出版信息

Biomed Res Int. 2013;2013:430290. doi: 10.1155/2013/430290. Epub 2013 Oct 28.

Abstract

The human induced pluripotent stem cells (hiPSCs) are derived from a direct reprogramming of human somatic cells to a pluripotent stage through ectopic expression of specific transcription factors. These cells have two important properties, which are the self-renewal capacity and the ability to differentiate into any cell type of the human body. So, the discovery of hiPSCs opens new opportunities in biomedical sciences, since these cells may be useful for understanding the mechanisms of diseases in the production of new diseases models, in drug development/drug toxicity tests, gene therapies, and cell replacement therapies. However, the hiPSCs technology has limitations including the potential for the development of genetic and epigenetic abnormalities leading to tumorigenicity. Nowadays, basic research in the hiPSCs field has made progress in the application of new strategies with the aim to enable an efficient production of high-quality of hiPSCs for safety and efficacy, necessary to the future application for clinical practice. In this review, we show the recent advances in hiPSCs' basic research and some potential clinical applications focusing on cancer. We also present the importance of the use of statistical methods to evaluate the possible validation for the hiPSCs for future therapeutic use toward personalized cell therapies.

摘要

人类诱导多能干细胞(hiPSCs)是通过外源表达特定转录因子,将人类体细胞直接重编程为多能状态而产生的。这些细胞具有两个重要特性,即自我更新能力和分化为人体任何细胞类型的能力。因此,hiPSCs 的发现为生物医学科学开辟了新的机会,因为这些细胞可用于在产生新的疾病模型、药物开发/药物毒性测试、基因治疗和细胞替代疗法中理解疾病的机制。然而,hiPSCs 技术存在局限性,包括可能发生遗传和表观遗传异常导致致瘤性。如今,hiPSCs 领域的基础研究在应用新策略方面取得了进展,目的是为了高效生产高质量的 hiPSCs,以确保未来在临床实践中的应用的安全性和有效性。在本文中,我们展示了 hiPSCs 基础研究的最新进展以及一些潜在的癌症临床应用。我们还介绍了使用统计方法评估 hiPSCs 未来治疗用途的个体化细胞治疗的可能验证的重要性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0e82/3830845/35ede6c5a664/BMRI2013-430290.001.jpg

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