title: mRNA as a versatile tool for exogenous protein expression.
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.
authors: TRON (Translational Oncology at the University Medical Center of the Johannes Gutenberg University Mainz), Mainz, Germany.
authors: Andreas N
authors: AN
authors: Kuhn
authors: Tim
authors: T
authors: Beiβert
authors: Petra
authors: P
authors: Simon
authors: Britta
authors: B
authors: Vallazza
authors: Janina
authors: J
authors: Buck
authors: Brian P
authors: BP
authors: Davies
authors: Ozlem
authors: O
authors: Tureci
authors: Ugur
authors: U
authors: Sahin
mesh_terms: D000818:Animals; D015870:Gene Expression; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D007113:Immunity, Innate; D011499:Protein Processing, Post-Translational; D020871:RNA Stability; D012333:RNA, Messenger / Q000235:genetics* / Q000276:immunology / Q000378:metabolism
chemical_list: D012333:RNA, Messenger