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Intracellular messenger RNAs (mRNAs) and their associated regulatory complexes, often termed messenger ribonucleoproteins (mRNPs), serve as the essential templates for protein synthesis and act as central hubs for post-transcriptional gene regulation (Dreyfuss et al., 2002, Nat Rev Mol Cell Biol). These complexes consist of mRNA molecules bound by a diverse array of RNA-binding proteins (RBPs) and non-coding RNAs that collectively determine the transcript's stability, cellular localization, and translation efficiency (Moore, 2005, Science). In various disease states, the dysregulation of specific mRNAs or the failure of their regulatory machinery can lead to the overproduction of oncogenic proteins or the deficiency of vital enzymes (Sahin et al., 2014, Nat Rev Drug Discov). Therapeutic interventions targeting these entities include antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) designed to degrade pathogenic transcripts or correct aberrant splicing (Crooke et al., 2018, Cell Metab). Additionally, synthetic mRNA technology allows for the intracellular delivery of functional transcripts to replace missing proteins or serve as antigenic templates for vaccines. While highly versatile, targeting intracellular RNA requires specialized delivery systems like lipid nanoparticles to ensure cellular uptake and protection from nucleases, while also managing potential immunogenic responses to exogenous nucleic acids.
RNA interference (RNAi), Antisense inhibition, Splice modulation, mRNA replacement, Translation inhibition
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