Messenger RNA of intended gene target (mRNA) (mRNA)

Messenger RNA (mRNA) serves as the critical intermediary in the central dogma of molecular biology, carrying genetic information from DNA to the ribosome for protein synthesis [1]. As a therapeutic target, mRNA allows for the modulation of protein expression levels, offering a pathway to treat diseases caused by 'undruggable' proteins that lack accessible binding pockets for traditional small molecules [2]. Drugs targeting mRNA, such as antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs), work by binding to specific sequences to trigger degradation or block translation [3]. This target class is instrumental in treating genetic disorders like spinal muscular atrophy and hereditary transthyretin-mediated amyloidosis by either silencing toxic genes or correcting splicing defects [4]. Small molecules are also being developed to target the secondary and tertiary structures of mRNA to influence its stability and translation efficiency [5]. While highly specific, mRNA-targeted therapies face challenges related to cellular delivery, stability in the bloodstream, and potential activation of the innate immune system [6]. The term 'mRNA of intended gene target' is a generic descriptor used in drug development to indicate that the therapeutic strategy focuses on the transcript of a specific, yet-to-be-named gene [7]. Effective utilization of this target requires precise knowledge of the target sequence to avoid off-target effects and ensure therapeutic efficacy [8].

Other names

Messenger RNAmRNATranscriptCoding RNAGene transcript

Drugs targeting mRNA typically function through RNA interference (RNAi), RNase H-mediated degradation, steric hindrance of translation, or modulation of alternative splicing [1][3].