Hepatic messenger RNA (mRNA) (mRNA)

Hepatic messenger RNA (mRNA) refers to the complete set of protein-coding RNA transcripts produced within the liver, primarily by hepatocytes [1]. While Hepatic mRNAs is a collective term rather than a single discrete target, specific mRNA sequences within this pool are the focus of a rapidly expanding class of nucleic acid therapeutics [2]. These therapies, including small interfering RNAs (siRNAs) and antisense oligonucleotides (ASOs), are designed to selectively bind to a target mRNA sequence to trigger its degradation or inhibit its translation into protein [1][3]. The liver is a particularly attractive site for these interventions due to its central role in synthesizing proteins involved in systemic diseases and the development of efficient delivery systems, such as N-acetylgalactosamine (GalNAc) conjugates, which target the asialoglycoprotein receptor on hepatocytes [2]. Approved drugs like Inclisiran and Patisiran demonstrate the clinical utility of targeting hepatic mRNAs to treat conditions such as hypercholesterolemia and hereditary transthyretin-mediated amyloidosis [3][4]. By silencing the production of pathogenic proteins at the source, these treatments offer a potent and durable alternative to traditional small molecule or monoclonal antibody therapies [1].

Other names

Liver mRNAHepatic transcriptomeHepatic mRNAsLiver messenger RNA

Drugs targeting hepatic mRNAs primarily utilize RNA interference (RNAi) or antisense oligonucleotide (ASO) technology to bind complementary sequences, leading to mRNA degradation via the RISC complex or RNase H, which subsequently prevents the translation of disease-associated proteins [1][2].