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Target intelligence / Profile preview
Huntingtin Gene mRNA (HTT mRNA)
- Target
- HTT mRNA
- Molecular classification
- mRNA, Non-coding RNA
01
Overview
The huntingtin gene mRNA is the messenger RNA transcribed from the HTT gene, which encodes the huntingtin protein. Expansion of the CAG repeat region in the HTT gene results in mutant HTT mRNA, which leads to the production of a toxic protein and Huntington's disease. Targeting mutant HTT mRNA is a therapeutic strategy for Huntington's disease.
- Other names
- HTT mRNAIT15 mRNA
02
Mechanism of action
Antisense oligonucleotide binding to mRNA, RNA interference
03
Biological functions
Template for protein synthesisAxonal transport (via Huntingtin protein)Vesicle trafficking (via Huntingtin protein)BDNF regulation (via Huntingtin protein)DNA repair (via Huntingtin protein)
04
Disease associations
Huntington's Disease
05
Safety considerations
Off-target effects of mRNA-targeting therapiesPotential impact on wild-type Huntingtin function
06
Biomarkers
HTT mRNA levelsCAG repeat length in HTT mRNA
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