LAUSR.org creates dashboard-style pages of related content for over 1.5 million academic articles. Sign Up to like articles & get recommendations!

Development of MTL-CEBPA: Small Activating RNA Drug for Hepatocellular Carcinoma

Photo by wulcan from unsplash

Background: Oligonucleotide drug development has revolutionised the drug discovery field. Within this field, ‘small’ or ‘short’ activating RNAs (saRNA) are a more recently discovered category of short double-stranded RNA with… Click to show full abstract

Background: Oligonucleotide drug development has revolutionised the drug discovery field. Within this field, ‘small’ or ‘short’ activating RNAs (saRNA) are a more recently discovered category of short double-stranded RNA with clinical potential. saRNAs promote transcription from target loci, a phenomenon widely observed in mammals known as RNA activation (RNAa). Objective: The ability to target a particular gene is dependent on the sequence of the saRNA. Hence, the potential clinical application of saRNAs is to increase target gene expression in a sequence-specific man-ner. saRNA-based therapeutics present opportunities for expanding the “druggable genome” with partic-ular areas of interest including transcription factor activation and cases of haploinsufficiency. Results and Conclusion: In this mini-review, we describe the pre-clinical development of the first saRNA drug to enter the clinic. This saRNA, referred to as MTL-CEBPA, induces increased expression of the transcription factor CCAAT/enhancer-binding protein alpha (CEBPα), a tumour suppressor and critical regulator of hepatocyte function. MTL-CEBPA is presently in Phase I clinical trials for hepatocel-lular carcinoma (HCC). The clinical development of MTL-CEBPA will demonstrate “proof of concept” that saRNAs can provide the basis for drugs which enhance target gene expression and consequently improve treatment outcome in patients

Keywords: drug; development mtl; mtl cebpa; carcinoma

Journal Title: Current Pharmaceutical Biotechnology
Year Published: 2018

Link to full text (if available)


Share on Social Media:                               Sign Up to like & get
recommendations!

Related content

More Information              News              Social Media              Video              Recommended



                Click one of the above tabs to view related content.