Department of Surgery, Weill Cornell Medicine, Cornell University, New York, New York, USA.
Stem Cells Transl Med. 2019 Aug;8(8):833-843. doi: 10.1002/sctm.18-0259. Epub 2019 Mar 19.
Chemically modified RNA (cmRNA) has potential as a safe and efficient tool for nucleic acid-based therapies and regenerative medicine. Modifications in the chemistry of mRNA can enhance stability, reduce immunogenicity, and thus facilitate mRNA-based nucleic acid therapy, which eliminates risk of insertional mutagenesis. In addition to these valuable advantages, the mRNA-based method showed significantly higher efficacy for reprogramming somatic cells to pluripotency compared with DNA- or protein-based methods. These findings suggest cmRNA can provide a powerful and safe tool for cell programming and reprogramming. Delivery methods, particularly using lipid nanoparticles, provide strategies for cell and organ-specific targeting. The present study comprehensively compares studies that have used cmRNAs for cell fate conversion and tissue engineering. The information should be useful for investigators looking to choose the most efficient and straightforward cmRNA-based strategy and protocol for tissue engineering and regenerative medicine research. Stem Cells Translational Medicine 2019;8:833&843.
化学修饰 RNA(cmRNA)作为核酸治疗和再生医学的安全有效的工具具有很大的潜力。mRNA 化学修饰可以提高稳定性、降低免疫原性,从而促进基于 mRNA 的核酸治疗,消除插入突变的风险。除了这些有价值的优势外,与基于 DNA 或蛋白质的方法相比,基于 mRNA 的方法在体细胞重编程为多能性方面显示出更高的功效。这些发现表明,cmRNA 可为细胞编程和重编程提供一种强大且安全的工具。递药方法,特别是使用脂质纳米颗粒,为细胞和器官特异性靶向提供了策略。本研究全面比较了使用 cmRNA 进行细胞命运转换和组织工程的研究。这些信息对于希望选择最有效和最简单的基于 cmRNA 的策略和方案用于组织工程和再生医学研究的研究人员来说应该是有用的。《Stem Cells Translational Medicine》2019 年;8:833&843.