Emergence of synthetic mRNA: In vitro synthesis of mRNA and its applications in regenerative medicine
- 주제(키워드) Synthetic mRNA , In vitro transcription (IVT) , Cellular reprograming , Regenerative medicine
- 주제(기타) Engineering, Biomedical; Materials Science, Biomaterials
- 설명문(일반) [Kwon, Hyokyoung; Kim, Minjeong; Seo, Yunmi; Moon, Yae Seul; Lee, Hwa Jeong; Lee, Kyuri; Lee, Hyukjin] Ewha Womans Univ, Grad Sch Pharmaceut Sci, Coll Pharm, Seoul 03760, South Korea
- 등재 SCIE, SCOPUS
- 발행기관 ELSEVIER SCI LTD
- 발행년도 2018
- URI http://www.dcollection.net/handler/ewha/000000150028
- 본문언어 영어
- Published As http://dx.doi.org/10.1016/j.biomaterials.2017.11.034
초록/요약
The field of gene therapy has evolved over the past two decades after the first introduction of nucleic acid drugs, such as plasmid DNA (pDNA). With the development of in vitro transcription (IVT) methods, synthetic mRNA has become an emerging class of gene therapy. IVT mRNA has several advantages over conventional pDNA for the expression of target proteins. mRNA does not require nuclear localization to mediate protein translation. The intracellular process for protein expression is much simpler and there is no potential risk of insertion mutagenesis. Having these advantages, the level of protein expression is far enhanced as comparable to that of viral expression systems. This makes IVT mRNA a powerful alternative gene expression system for various applications in regenerative medicine. In this review, we highlight the synthesis and preparation of IVT mRNA and its therapeutic applications. The article includes the design and preparation of IVT mRNA, chemical modification of IVT mRNA, and therapeutic applications of IVT mRNA in cellular reprogramming, stem cell engineering, and protein replacement therapy. Finally, future perspectives and challenges of IVT mRNA are discussed. (C) 2017 Elsevier Ltd. All rights reserved.
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