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基于 RNA 的核重编程和谱系转换工具:迈向临床应用。

RNA-based tools for nuclear reprogramming and lineage-conversion: towards clinical applications.

机构信息

Cardiovascular Development and Repair Department, Centro Nacional de Investigaciones Cardiovasculares (CNIC), Melchor Fernández Almagro 3, 28029, Madrid, Spain,

出版信息

J Cardiovasc Transl Res. 2013 Dec;6(6):956-68. doi: 10.1007/s12265-013-9494-8. Epub 2013 Jul 13.

Abstract

The therapeutic potential of induced pluripotent stem cells (iPSCs) is well established. Safety concerns remain, however, and these have driven considerable efforts aimed at avoiding host genome alteration during the reprogramming process. At present, the tools used to generate human iPSCs include (1) DNA-based integrative and non-integrative methods and (2) DNA-free reprogramming technologies, including RNA-based approaches. Because of their combined efficiency and safety characteristics, RNA-based methods have emerged as the most promising tool for future iPSC-based regenerative medicine applications. Here, I will discuss novel recent advances in reprogramming technology, especially those utilizing the Sendai virus (SeV) and synthetic modified mRNA. In the future, these technologies may find utility in iPSC reprogramming for cellular lineage-conversion, and its subsequent use in cell-based therapies.

摘要

诱导多能干细胞(iPSCs)的治疗潜力已得到充分证实。然而,安全性问题仍然存在,这促使人们做出了巨大努力,旨在避免在重编程过程中宿主基因组发生改变。目前,用于生成人类 iPSCs 的工具包括(1)基于 DNA 的整合和非整合方法,以及(2)无 DNA 的重编程技术,包括基于 RNA 的方法。由于其兼具高效性和安全性,基于 RNA 的方法已成为未来基于 iPSC 的再生医学应用中最有前途的工具。在这里,我将讨论重编程技术的最新进展,特别是利用仙台病毒(SeV)和合成修饰的 mRNA 的方法。在未来,这些技术可能在 iPSC 重编程用于细胞谱系转换及其随后在细胞治疗中的应用中找到用途。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7f7b/3838600/5d652affc75b/12265_2013_9494_Fig1_HTML.jpg

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