European Journal of Neurology
Huntington's disease: a clinical review
What this article backs
- Htt Gene Mutation Mutant Huntingtin Protein
- Mutant Huntingtin Protein Neurodegeneration
- Htt Lowering Therapy Mutant Huntingtin Protein
- Antisense Oligonucleotide Mutant Huntingtin Protein
- Htt Gene Mutation enables Mutant Huntingtin Protein.
- Mutant Huntingtin Protein triggers Neurodegeneration.
- Htt Lowering Therapy decreases Mutant Huntingtin Protein.
- Antisense Oligonucleotide decreases Mutant Huntingtin Protein.
In its own words
mutant huntingtin results in neuronal dysfunction and death through a number of mechanisms, including disruption of proteostasis, transcription and mitochondrial function and direct toxicity of the mutant protein
Antisense oligonucleotide therapy is one such approach with clinical trials currently under way. This may bring us one step closer to treating and potentially preventing this devastating condition.
a fully penetrant neurodegenerative disease caused by a dominantly inherited CAG trinucleotide repeat expansion in the huntingtin gene
Perhaps the most promising of these are the emerging therapies aimed at lowering levels of mutant huntingtin.
