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Muscleblind-like Splicing Regulator 1 (None)
- Target
- None
- Molecular classification
- RNA-binding protein, Splicing regulator, Transcription factor
01
Overview
Muscleblind-like Splicing Regulator 1 (MBNL1) is an RNA-binding protein that regulates alternative pre-mRNA splicing. It acts as both a repressor and activator, influencing muscle differentiation and mRNA maturation. In myotonic dystrophy type 1 (DM1), expanded CUG repeats sequester MBNL1, leading to mis-splicing and disease pathology. MBNL1 recognizes YGCY motifs and regulates exon inclusion/exclusion. Aberrant splicing patterns are seen in DM patients due to the loss or sequestration of functional MBNL1.
- Other names
- MBNL1
02
Mechanism of action
Binds to YGCY motifs in pre-mRNAs to regulate exon inclusion or exclusion; Sequestration by CUG repeats in DM1
03
Biological functions
Alternative pre-mRNA splicing regulationRegulation of gene expressionMuscle cell differentiationmRNA maturation
04
Disease associations
Myotonic dystrophy type 1 (DM1)Cancer
05
Safety considerations
Off-target effects on splicingPotential for dysregulation of other cellular processes
06
Biomarkers
MBNL1 expression levelsSplicing patterns of MBNL1 target genes
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