mRNA as a Versatile Tool for Exogenous Protein Expression

<!DOCTYPE html> <html lang="en"> <head><script type="text/javascript" src="https://web-static.archive.org/_static/js/bundle-playback.js?v=7YQSqjSh" charset="utf-8"></script> <script type="text/javascript" src="https://web-static.archive.org/_static/js/wombat.js?v=txqj7nKC" charset="utf-8"></script> <script>window.RufflePlayer=window.RufflePlayer||{};window.RufflePlayer.config={"autoplay":"on","unmuteOverlay":"hidden"};</script> <script type="text/javascript" src="https://web-static.archive.org/_static/js/ruffle/ruffle.js"></script> <script type="text/javascript"> __wm.init("https://web.archive.org/web"); __wm.wombat("http://www.eurekaselect.com/article/45604","20220615180724","https://web.archive.org/","web","https://web-static.archive.org/_static/", "1655316444"); </script> <link rel="stylesheet" type="text/css" href="https://web-static.archive.org/_static/css/banner-styles.css?v=p7PEIJWi" /> <link rel="stylesheet" type="text/css" href="https://web-static.archive.org/_static/css/iconochive.css?v=3PDvdIFv" />  <meta charset="utf-8"> <title> mRNA as a Versatile Tool for Exogenous Protein Expression | Bentham Science </title> <meta content="IE=edge" http-equiv="X-UA-Compatible"> <meta content="width=device-width, initial-scale=1" name="viewport">  <meta content="Wy5iQJS98lhvb9aQ31VLzULd8psaH8WsuveGyc6p" name="csrf-token">  <meta name="description" content="Several viral and non-viral vectors have been developed for exogenous protein expression in specific cells. Conventionally, this purpose is achieved throu"/> <meta name="keywords" content="mRNA as a Versatile Tool for Exogenous Protein Expression"> <meta property="og:type" content="website"/> <meta property="og:title" content="mRNA as a Versatile Tool for Exogenous Protein Expression"/> <meta property="og:url" content="https://web.archive.org/web/20220615180724/https://www.eurekaselect.com/article/45604"/> <meta property="og:image" content="https://web.archive.org/web/20220615180724im_/http://www.eurekaselect.com/images/bentham/CGT_0.gif"/> <meta property="og:description" content="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."/> <meta property="og:site_name" content="http://www.eurekaselect.com"/> <meta name="citation_title" content="mRNA as a Versatile Tool for Exogenous Protein Expression"/> <meta name="citation_author" content="Andreas N. Kuhn"/> <meta name="citation_author" content="Tim Beißert"/> <meta name="citation_author" content="Petra Simon"/> <meta name="citation_author" content="Britta Vallazza"/> <meta name="citation_author" content="Janina Buck"/> <meta name="citation_author" content="Brian P. Davies"/> <meta name="citation_author" content="Ozlem Tureci"/> <meta name="citation_author" content="Ugur Sahin"/> <meta name="citation_publish_on" content="2012-09-04 "/> <meta name="citation_journal_title" content="Current Gene Therapy"> <meta name="citation_volume" content="12"> <meta name="citation_issue" content="5"> <meta name="citation_firstpage" content="347"> <meta name="citation_lastpage" content="361"> <meta name="citation_pdf_url" content="http://www.eurekaselect.com/article/45604"> <script defer="" src="https://web.archive.org/web/20220615180724js_/http://www.eurekaselect.com/js/app.js"></script> <script type="text/javascript" src="https://web.archive.org/web/20220615180724js_/http://www.eurekaselect.com/js/jquery.min.js"></script>  <script src="https://web.archive.org/web/20220615180724js_/https://kit.fontawesome.com/1cb4e2d48a.js" crossorigin="anonymous"></script>   <link 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class="col-md-5"> <div class="row"> <div class="col-12"> <div class="media"> <a href="/web/20220615180724/http://www.eurekaselect.com/journal/28/current-issue"> <img class="mr-3 img-thumbnail" src="/web/20220615180724im_/http://www.eurekaselect.com/images/bentham/CGT_0.gif" alt="Generic placeholder image"> </a> <div class="media-body"> <h2 class="pr-lg-3"> Current Gene Therapy </h2> <a tabindex="0" class="btn btn-outline-light btn-sm mb-3" role="button" data-toggle="popover" data-html="true" data-trigger="hover" data-content="<p><strong>Editor-in-Chief:<br></strong><b></b>Liang Cheng<b><i></i></b><br>College of Bioinformatics Science and Technology<br>Harbin Medical University<br>Harbin, Heilongjiang<br>China<br></p>">Editor-in-Chief <i class="fas fa-angle-double-right"></i></a> <p>ISSN (Print): 1566-5232<br> ISSN (Online): 1875-5631</p> </div> </div> </div> <div class="col-md-12 mt-3"> <a href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/" class="btn 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Kuhn, Tim Beißert, Petra Simon, Britta Vallazza, Janina Buck, Brian P. Davies, Ozlem Tureci and <a href="javascript:void(0)" class="text-secondary" title="Affiliation" data-html="true" data-toggle="popover" data-placement="bottom" data-trigger="hover" data-content="<ul><li>TRON (Translational Oncology at the University Medical Center of the Johannes Gutenberg University Mainz), Langenbeckstr. 1, 55131 Mainz, Germany.,Germany </li></ul>">Ugur Sahin</a> </span> <div class="row"> <div class="col-md-8"> <p>Volume 12, Issue 5, 2012 </p>  <p><strong>Page:</strong> [347 - 361] <span class="float-right"><strong>Pages:</strong> 15 </span></p> <p><strong>DOI:</strong> <a href="https://web.archive.org/web/20220615180724/http://dx.doi.org/10.2174/156652312802762536" class="btn-link">10.2174/156652312802762536</a></p> <p><strong>Price:</strong> $65</a></p> </div> <form method="POST" action="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/cart" accept-charset="UTF-8" id="addtocartForm"><input name="_token" type="hidden" value="Wy5iQJS98lhvb9aQ31VLzULd8psaH8WsuveGyc6p"> <input type="hidden" name="param" value="eyJpdiI6IjNzUXFQMFNNUms5bFp4K2VZMmpwWFE9PSIsInZhbHVlIjoiK0VWVnZEQXdEblFyNEtPdnZCekJnUT09IiwibWFjIjoiYzY5YjE2OTYxYzU4OWQ2YTk3ZTM4YzU1NDE1MzJhNTU0NTAyMmYwMDRmNzEwZGNiY2U2ZDVjMmEwNGY4MmYxZCIsInRhZyI6IiJ9"> <div class="col-md-4 text-right"> <button type="submit" id="addcartbtn" class="btn btn-download hvr-icon-hang">Purchase<br>PDF<br><i class="fas fa-cart-plus hvr-icon"></i></button> </div> </form> </div> </div> </div> </div> </section>   <main id="article"> <section> <div class="container"> <div class="row"> <div class="col-md-8">   <div id="abstract" class="card bigbox-hvr-slow"> <div class="card-header"> <h5>Abstract</h5> </div> <div class="card-body"> <p class="text-justify"> 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. </p> <p><strong>Keywords: </strong> <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue=Gene therapy">Gene therapy</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= genetic modifications"> genetic modifications</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= immunotherapy"> immunotherapy</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= in vitro transcription"> in vitro transcription</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= mRNA"> mRNA</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= RNA stability"> RNA stability</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= stem cells"> stem cells</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue=T-cell therapy"> T-cell therapy</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= transfection"> transfection</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= translation"> translation</a> </p> </div> </div> <div class="card-body"> <a href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/article/45603" class="float-left btn btn-primary btn-sm active" data-mdb-toggle="button" autocomplete="off" aria-pressed="true">« Previous</a> <a href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/article/45605" class="float-right btn btn-primary btn-sm active" data-mdb-toggle="button" autocomplete="off" aria-pressed="true">Next »</a> </div> <br>  <div class="row mx-0"> <div class="col-lg-auto download-bar text-sm-center p-2 mr-md-3 bg-siteblue rounded"> <div class="btn-group"> <form method="POST" action="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/marklist" accept-charset="UTF-8" id="markItemForm"><input name="_token" type="hidden" value="Wy5iQJS98lhvb9aQ31VLzULd8psaH8WsuveGyc6p"> <input type="hidden" name="param" value="eyJpdiI6IlB5QWJ4RnZQcFd1U05ZSFlBdVN4dlE9PSIsInZhbHVlIjoiOStJVGFmVEJGNTNCOU42aCtMWVJKUT09IiwibWFjIjoiMjI5OGIzN2Q0M2FhMzg2OWY2YmMzNzYzYmJjNTY4ODJjMjM2ZDA3NzE2NWJmNTAxMzYyN2M4MjIyODBjMjA1MiIsInRhZyI6IiJ9"> <input type="hidden" name="itemtype" value="1"> <button type="submit" id="markitembtn" class="btn btn-light bg-white btn-sm">Mark Item</button> </form> </div> <div class="btn-group"> <form method="POST" action="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/cart" accept-charset="UTF-8" id="addtocartForm"><input name="_token" type="hidden" value="Wy5iQJS98lhvb9aQ31VLzULd8psaH8WsuveGyc6p"> <input type="hidden" name="param" value="eyJpdiI6Ik00MGsvQUNCKzJZczhMZS9XTk1Xdnc9PSIsInZhbHVlIjoiVS9BL2x6QWdCWmlYYkhOWnNHKzZpZz09IiwibWFjIjoiMjExMDU1NzQ1MTE0MWE0ZDM0N2FmN2IwZTZjYjc3ZTUzMjg4ZGMzNGU1NzYyMDg3ZWQ0YmY5NjAyNzUyNGQ2ZiIsInRhZyI6IiJ9"> <button type="submit" id="addcartbtn" class="btn btn-light bg-white btn-sm">Purchase PDF</button> </form> </div> <a class="btn btn-light bg-white btn-sm" href="javascript:void(0);" id="RightsLinkPopUp">Rights & Permissions</a> <a class="btn btn-light bg-white btn-sm" href="javascript:void(0);" data-toggle="modal" data-target="#PrintWindow">Print</a> <a class="btn btn-light bg-white btn-sm" href="javascript:void(0);" data-toggle="modal" data-target="#ExportWindow">Export</a> <a class="btn btn-light bg-white btn-sm" href="javascript:void(0);" data-toggle="modal" data-target="#CiteWindow">Cite as</a> </div>  <div class="modal fade" id="PrintWindow" role="dialog"> <div class="modal-dialog modal-lg"> <div class="modal-content"> <div class="modal-header"> <h4 class="modal-title" id="modalLabelprimary">Current Gene Therapy</h4> </div> <div class="modal-body "> <p><strong>Title:</strong>mRNA as a Versatile Tool for Exogenous Protein Expression</p> <p><strong>Volume:</strong> 12 <strong>Issue:</strong> 5 </p> <p><strong>Author(s): </strong>Andreas N. Kuhn, Tim Beißert, Petra Simon, Britta Vallazza, Janina Buck, Brian P. Davies, Ozlem Tureci and Ugur Sahin</p> <p><strong>Affiliation: </strong><ul><li>TRON (Translational Oncology at the University Medical Center of the Johannes Gutenberg University Mainz), Langenbeckstr. 1, 55131 Mainz, Germany.,Germany </li></ul></p> <p><strong>Keywords:</strong> <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue=Gene therapy">Gene therapy</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= genetic modifications"> genetic modifications</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= immunotherapy"> immunotherapy</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= in vitro transcription"> in vitro transcription</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= mRNA"> mRNA</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= RNA stability"> RNA stability</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= stem cells"> stem cells</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue=T-cell therapy"> T-cell therapy</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= transfection"> transfection</a>, <a target="_blank" class="btn-link" href="https://web.archive.org/web/20220615180724/http://www.eurekaselect.com/search?searchvalue= translation"> translation</a> </p> <p><strong>Abstract:</strong> 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. 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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) . <a href="https://web.archive.org/web/20220615180724/https://dx.doi.org/10.2174/156652312802762536">https://dx.doi.org/10.2174/156652312802762536</a> </p> <table class="table"> <tr> <td><b>DOI</b><br><a href="https://web.archive.org/web/20220615180724/https://dx.doi.org/10.2174/156652312802762536">https://dx.doi.org/10.2174/156652312802762536</a></td> <td><b>Print ISSN</b><br>1566-5232</td> </tr> <tr> <td><b>Publisher Name</b><br>Bentham Science Publisher</td> <td><b>Online ISSN</b><br>1875-5631</td> </tr> </table> </div> <div class="modal-footer"> <button type="button" class="btn btn-outline-dark" data-dismiss="modal">Close</button> <a class="btn btn-outline-primary" href="/web/20220615180724/http://www.eurekaselect.com/journal/28/about-journal" target="_blank">About this journal</a> </div> </div> </div> </div>  <div class="col pl-md-0 mt-xs-2 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mRNA as a Versatile Tool for Exogenous Protein Expression | Bentham Science