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Stathmin 2 mRNA splicing
- Molecular classification
- mRNA splicing, RNA splicing factor
01
Overview
Stathmin 2 (STMN2) mRNA splicing is a critical process regulated by TDP-43. Aberrant splicing, particularly cryptic exon inclusion, leads to truncated, non-functional STMN2 protein and is implicated in neurodegenerative diseases such as ALS and FTD. Therapeutic strategies aim to restore proper STMN2 splicing using ASOs or RNA-targeted CRISPR effectors.
- Other names
- STMN2 mRNA splicingSCG10 mRNA splicing
02
Mechanism of action
Antisense oligonucleotides bind near cryptic splice sites to block aberrant exon inclusion and restore full-length transcript. RNA-targeted CRISPR effectors block access to cryptic splice/polyA sites via steric hindrance.
03
Biological functions
Regulation of mRNA splicingModulation of stathmin-2 expressionRegulation of axonal growth and regeneration
04
Disease associations
Amyotrophic lateral sclerosis (ALS)Frontotemporal lobar degeneration/dementia (FTLD)Alzheimer's diseaseNeurodegenerative disease
05
Safety considerations
Off-target effects of ASOsPotential immunogenicity of ASOsDelivery challenges to the central nervous systemSpecificity of CRISPR effectors
06
Interacting drugs
Antisense Oligonucleotides (ASOs)
1 more in the full profile.
07
Biomarkers
STMN2 mRNA levelsCryptic exon inclusion in STMN2 mRNATDP-43 expression and localization
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