Abstract
Several viral and non-viral vectors have been developed for exogenous protein expression in specific cells. Conventionally, this purpose is achieved through the use of recombinant DNA. But mainly due to the risks associated with permanent genetic alteration of cells, safety and ethical concerns have been raised for the use of DNA-based vectors in human clinical therapy. In the last years, synthetic messenger RNA has emerged as powerful tool to deliver genetic information. RNA vectors exhibit several advantages compared to DNA and are particularly interesting for applications that require transient gene expression. RNA stability and translation efficiency can be increased by cis-acting structural elements in the RNA such as the 5’-cap, the poly(A)-tail, untranslated regions and the sequence of the coding region. Here we review recent developments in the optimization of messenger RNA as vector for modulation of protein expression emphasizing on stability, transfection and immunogenicity. In addition, we summarize current pre-clinical and clinical studies using RNA-based vectors for immunotherapy, T cell, stem cell as well as gene therapy.
Keywords: Gene therapy, genetic modifications, immunotherapy, in vitro transcription, mRNA, RNA stability, stem cells, T-cell therapy, transfection, translation
Current Gene Therapy
Title:mRNA as a Versatile Tool for Exogenous Protein Expression
Volume: 12 Issue: 5
Author(s): Andreas N. Kuhn, Tim Beißert, Petra Simon, Britta Vallazza, Janina Buck, Brian P. Davies, Ozlem Tureci and Ugur Sahin
Affiliation:
Keywords: Gene therapy, genetic modifications, immunotherapy, in vitro transcription, mRNA, RNA stability, stem cells, T-cell therapy, transfection, translation
Abstract: Several viral and non-viral vectors have been developed for exogenous protein expression in specific cells. Conventionally, this purpose is achieved through the use of recombinant DNA. But mainly due to the risks associated with permanent genetic alteration of cells, safety and ethical concerns have been raised for the use of DNA-based vectors in human clinical therapy. In the last years, synthetic messenger RNA has emerged as powerful tool to deliver genetic information. RNA vectors exhibit several advantages compared to DNA and are particularly interesting for applications that require transient gene expression. RNA stability and translation efficiency can be increased by cis-acting structural elements in the RNA such as the 5’-cap, the poly(A)-tail, untranslated regions and the sequence of the coding region. Here we review recent developments in the optimization of messenger RNA as vector for modulation of protein expression emphasizing on stability, transfection and immunogenicity. In addition, we summarize current pre-clinical and clinical studies using RNA-based vectors for immunotherapy, T cell, stem cell as well as gene therapy.
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N. Kuhn Andreas, Beißert Tim, Simon Petra, Vallazza Britta, Buck Janina, P. Davies Brian, Tureci Ozlem and Sahin Ugur, mRNA as a Versatile Tool for Exogenous Protein Expression, Current Gene Therapy 2012; 12 (5) . https://dx.doi.org/10.2174/156652312802762536
DOI https://dx.doi.org/10.2174/156652312802762536 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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