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Target intelligence / Profile preview
Frataxin Gene Expanded GAA Repeat (FXN GAA Repeat)
- Target
- FXN GAA Repeat
- Molecular classification
- Non-coding DNA, Intronic Repeat Expansion
01
Overview
The expanded GAA repeat in the first intron of the FXN gene is the primary cause of Friedreich ataxia. This expansion leads to transcriptional repression of the FXN gene, resulting in reduced frataxin protein levels and subsequent mitochondrial dysfunction. The length of the GAA repeat correlates with disease severity.
- Other names
- FXN GAA expansionFriedreich ataxia GAA repeatFrataxin intron 1 GAA repeat
02
Mechanism of action
N/A (Target is a non-coding region of DNA. Therapies aim to upregulate FXN transcription or compensate for frataxin deficiency)
03
Biological functions
Regulation of gene transcriptionChromatin structureMitochondrial function (indirectly via frataxin)
04
Disease associations
Friedreich ataxia
05
Safety considerations
Delivery of therapeutic agents to the nucleusSpecificity of targeting the expanded repeat without affecting normal allelesOff-target effects of gene editing or transcriptional activation strategies
06
Biomarkers
GAA repeat lengthFrataxin protein levelsFXN mRNA levels
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