Precursor messenger RNA of disease-causing gene (pre-mRNA)

Precursor messenger RNA (**pre-messenger RNA** or **pre-mRNA**) is a single-stranded nucleic acid molecule transcribed from DNA that contains both exons and introns. It serves as the immediate product of transcription before undergoing processing steps such as 5' capping, polyadenylation, and most importantly, **splicing**, which removes intronic sequences to produce mature messenger RNA (**mRNA**). This mature mRNA then serves as a template for protein synthesis during translation. Disruption in pre-mRNA processing—especially errors in splicing—can lead to various human diseases. Approximately 15% of mutations causing genetic disorders affect pre‐mRNA splicing signals or regulatory elements within disease-causing genes. Such disruptions are implicated in conditions like cystic fibrosis and retinitis pigmentosa among others[1][2][4]. While drugs do not typically target pre-mRNAs directly, therapeutic strategies often focus on modulating their processing using antisense oligonucleotides or small molecules that influence spliceosome activity.

Other names

Pre-mRNAPrimary transcript RNAHeterogeneous nuclear RNA (hnRNA)

Therapeutics may act by modifying splice site selection or correcting aberrant splicing through antisense oligonucleotides or small molecules targeting the spliceosome.