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Huntingtin mRNA (HTT mRNA)
- Target
- HTT mRNA
- Molecular classification
- mRNA, RNA
01
Overview
Huntingtin mRNA is the messenger RNA that encodes the huntingtin (HTT) protein, which plays a crucial role in various cellular functions and is associated with Huntington's disease (HD) when mutated. It contains a CAG repeat sequence, which, when expanded, leads to the production of mutant HTT protein and the development of HD. Targeting HTT mRNA is a therapeutic strategy for treating Huntington's disease.
- Other names
- mHTT mRNA (mutant Huntingtin mRNA)HTT transcript
02
Mechanism of action
Antisense oligonucleotides bind to HTT mRNA and promote its degradation. RNA interference uses small interfering RNAs to target HTT mRNA for degradation. Zinc-Finger Transcriptional Repressors aim to reduce transcription of the HTT gene. CRISPR/Cas9 methods also aim to reduce transcription by targeting the DNA directly.
03
Biological functions
Encodes Huntingtin proteinRegulation of gene expressionPost-transcriptional regulation
04
Disease associations
Huntington's DiseaseNeurodegenerative disease
05
Safety considerations
Off-target effects of ASOs and RNAiImmunogenicity of ASOs and RNAiPotential for incomplete HTT loweringUnintended consequences of reducing HTT expression
06
Interacting drugs
Antisense Oligonucleotides (ASOs)
3 more in the full profile.
07
Biomarkers
HTT mRNA levels in blood or CSFCAG repeat length in HTT gene
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