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Abstract
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.