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Precursor messenger RNA (pre-mRNA)
- Target
- pre-mRNA
- Molecular classification
- Other (RNA substrate; non-coding/intermediate form of messenger RNA)
01
Overview
Precursor messenger RNA (pre-mRNA) is a single-stranded RNA molecule synthesized from DNA by RNA polymerase II. It undergoes several key modifications—5′ capping, 3′ polyadenylation, and splicing—to become mature mRNA for translation. The splicing process removes non-coding introns and joins coding exons to enable proper protein synthesis. Pre-mRNA structure and associated modifications play crucial roles in regulating gene expression and protein diversity. Aberrant pre-mRNA processing and splicing are implicated in a range of human diseases, most notably cancer. While pre-mRNA itself is not a classical drug target, several therapeutic approaches aim to modulate its processing to treat genetic and acquired diseases[1][2][4][5][6][10].
- Other names
- pre-mRNAmRNA precursor
02
Mechanism of action
Modifying splicing patterns by binding to pre-mRNA or spliceosomal components Exon inclusion/exclusion adjustment in therapeutic settings
03
Biological functions
Template for post-transcriptional splicingRNA processing including 5′ capping, 3′ polyadenylation, and intron removalRegulation of gene expressionGeneration of protein diversity through alternative splicingInteraction with RNA-binding proteins and spliceosomal complexes
04
Disease associations
Cancer (aberrant splicing isoforms can drive proliferation, metastasis, and apoptosis dysregulation)Neurodegenerative disease (defects in splicing implicated in pathological RNA processing)Other (rare congenital splicing disorders, some viral infections exploit host pre-mRNA splicing machinery)
05
Safety considerations
Off-target effects of splice modulators (may alter splicing in unintended genes)Delivery and specificity for RNA-targeted drugs (nucleic acid drugs may be unstable or elicit immune responses)Risk of disrupting essential housekeeping gene splicing
06
Interacting drugs
Splice modulating drugs (e.g., antisense oligonucleotides, small molecules such as risdiplam and nusinersen for spinal muscular atrophy, which modulate pre-mRNA splicing)
1 more in the full profile.
07
Biomarkers
Aberrant or alternative splicing isoforms can serve as biomarkers for diseases such as certain cancers (e.g., detection of specific oncogenic splice variants)Mutational signatures affecting splice sites in pre-mRNA
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