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Huntingtin Gene Messenger RNA (HTT mRNA)
- Target
- HTT mRNA
- Molecular classification
- mRNA, Transcript
01
Overview
The huntingtin gene messenger RNA (HTT mRNA) is the transcript produced from the HTT gene, which provides the instructions for synthesizing the huntingtin protein. This mRNA acts as a template that ribosomes read to assemble amino acids into the huntingtin protein. Huntington’s disease (HD) results from an expanded CAG trinucleotide repeat within exon 1 of the HTT gene, leading to mutant HTT mRNA encoding an abnormally long polyglutamine tract in huntingtin, resulting in toxic gain-of-function effects. HTT mRNA represents a validated target for novel therapeutics aiming at reducing pathogenic protein production via direct transcript targeting.
- Other names
- HTT transcriptHuntingtin mRNA
02
Mechanism of action
Reduction of mutant HTT mRNA levels, preventing synthesis of toxic mutant Huntingtin protein.
03
Biological functions
Template for protein synthesisAxonal transport (indirectly via Huntingtin protein)Vesicle trafficking and endocytosis (indirectly via Huntingtin protein)Cytoskeletal anchoring (indirectly via Huntingtin protein)Regulation of brain-derived neurotrophic factor (BDNF) expression (indirectly via Huntingtin protein)Protection against apoptosis (indirectly via Huntingtin protein)DNA repair (potentially, indirectly via Huntingtin protein)
04
Disease associations
Huntington's DiseaseNeurodegenerative disease
05
Safety considerations
Off-target effects of gene silencing therapiesPotential disruption of wild-type HTT functionDelivery challenges to the brainImmunogenicity of therapeutic agents
06
Interacting drugs
Antisense oligonucleotides (ASOs)
2 more in the full profile.
07
Biomarkers
CAG repeat length in HTT geneHTT mRNA levelsMutant Huntingtin protein levels
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