Disease-associated messenger RNA (mRNA) (mRNA)

Messenger RNA (mRNA) is a single-stranded molecule that conveys genetic information from DNA to the ribosome, where it specifies the amino acid sequence of the protein products of gene expression (Sahin et al., 2014, Nature Reviews Drug Discovery). In many diseases, mRNA sequences are altered through mutations, dysregulated expression, or aberrant splicing, contributing to the pathogenesis of cancer, genetic disorders, and viral infections (Crooke et al., 2018, Cell Metabolism). Therapeutic strategies targeting mRNA include antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs), which can degrade target mRNA or modulate its splicing to restore or inhibit protein function (Damase et al., 2021, Frontiers in Bioengineering and Biotechnology). Furthermore, mRNA-based vaccines and replacement therapies utilize synthetic mRNA to instruct cells to produce specific antigens or therapeutic proteins (Kowalski et al., 2019, Molecular Therapy). This approach is particularly valuable for targeting proteins that are considered "undruggable" by traditional small molecules or antibodies (Weng et al., 2020, Signal Transduction and Targeted Therapy).

Other names

mRNAMessenger RNATranscriptCoding RNADisease-associated mRNA

RNA interference (RNAi) via RISC-mediated cleavage, Antisense oligonucleotide (ASO) mediated RNase H degradation, Splice-switching to induce exon skipping or inclusion, and mRNA-based translation for protein replacement or immunization.