Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases

Trepotec, Zeljka; Lichtenegger, Eva; Plank, Christian; Aneja, Manish K. und Rudolph, Carsten (2019): Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases. In: Molecular Therapy, Bd. 27, Nr. 4: S. 794-802

Abstract

Promising improvements in the field of transcript therapeutics have clearly enhanced the potential of mRNA as a new pillar for protein replacement therapies. Synthetic mRNAs are engineered to replace mutated mRNAs and to be immunologically inconspicuous and highly stable while maximizing protein expression. Approaches to deliver mRNA into the cellular cytoplasm safely and efficiently have been further developed so that two mRNA-based approaches replacing vascular endothelial growth factor (VEGF) and cystic fibrosis transmembrane conductance regulator (CFTR) have now made it into clinical trials. These studies bring mRNA therapeutics for protein replacement therapy closer to clinical realization. Herein, we provide an overview of preclinical and clinical developments of mRNA therapeutics for liver diseases.

• Dokumententyp: Zeitschriftenartikel
• Fakultät: Medizin
• Themengebiete: 600 Technik, Medizin, angewandte Wissenschaften > 610 Medizin und Gesundheit
• ISSN: 1525-0016
• Sprache: Englisch