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Target intelligence / Profile preview
GAA Repeat Expansion in FXN Gene (GAA Expansion (FXN))
- Target
- GAA Expansion (FXN)
- Molecular classification
- Non-coding DNA sequence, Intronic repeat, Epigenetic regulator (indirectly)
01
Overview
The GAA repeat expansion in the FXN gene refers to an abnormal increase in the number of consecutive GAA trinucleotide repeats located within intron 1 of the frataxin (FXN) gene. This expansion leads to transcriptional silencing of FXN, resulting in reduced frataxin protein levels and Friedreich's ataxia (FRDA).
- Other names
- FXN GAA repeatFriedreich's ataxia GAA expansionFrataxin GAA repeat expansionFXN Intron 1 GAA repeat
02
Mechanism of action
N/A
03
Biological functions
Regulation of FXN gene expressionTranscriptional silencingChromatin modificationMaintenance of genome stability (indirectly)Frataxin protein production regulation
04
Disease associations
Friedreich's ataxia (FRDA)Neurodegenerative diseaseMitochondrial dysfunction
05
Safety considerations
Off-target effects of therapies aimed at correcting the expansionChallenges in delivering therapies to the affected tissues (e.g., CNS)Potential for somatic instability to complicate therapeutic outcomes
06
Biomarkers
GAA repeat lengthFrataxin protein levelsFXN mRNA levelsHistone methylation patterns at the FXN locus
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