Current Gene Therapy

Ignacio Anegon
Director INSERM UMR 1064-Center for Research in Transplantation and Immunology
CHU de Nantes. 30, boulevard
Nantes
France

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mRNA as a Versatile Tool for Exogenous Protein Expression

Author(s): Andreas N. Kuhn, Tim Beißert, Petra Simon, Britta Vallazza, Janina Buck, Brian P. Davies, Ozlem Tureci, Ugur Sahin.

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

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Article Details

VOLUME: 12
ISSUE: 5
Year: 2012
Page: [347 - 361]
Pages: 15
DOI: 10.2174/156652312802762536
Price: $58