6533b855fe1ef96bd12b0077
RESEARCH PRODUCT
mRNA as a versatile tool for exogenous protein expression.
ÖZlem TüreciUgur SahinAndreas KuhnJanina BuckBrian P. DaviesPetra SimonBritta VallazzaTim Beißertsubject
RNA StabilityGenetic VectorsGene ExpressionComputational biologyBiologySmall hairpin RNADrug DiscoveryGene expressionGeneticsAnimalsHumansVector (molecular biology)RNA MessengerMolecular BiologyPost-transcriptional regulationGenetics (clinical)GeneticsMessenger RNAGene Transfer TechniquesRNAGenetic TherapyImmunity InnateRNA silencingRegulatory sequenceMolecular MedicineProtein Processing Post-Translationaldescription
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.
| year | journal | country | edition | language |
|---|---|---|---|---|
| 2012-09-01 | Current gene therapy |