Molecular Therapy
Potent and Selective Antisense Oligonucleotides Targeting Single-Nucleotide Polymorphisms in the Huntington Disease Gene / Allele-Specific Silencing of Mutant Huntingtin
What this article backs
- Htt Gene Mutation Mutant Huntingtin Protein
- Mutant Huntingtin Protein Neurodegeneration
- Antisense Oligonucleotide Mutant Huntingtin Protein
- Wild Type Huntingtin Protein Neurodegeneration
- Cet Modification Antisense Oligonucleotide
- Htt Gene Mutation triggers Mutant Huntingtin Protein.
- Mutant Huntingtin Protein triggers Neurodegeneration.
- Antisense Oligonucleotide decreases Mutant Huntingtin Protein.
- Wild Type Huntingtin Protein enables Neurodegeneration.
- Cet Modification increases Antisense Oligonucleotide.
In its own words
wild-type HTT protein is essential for development and has critical roles in maintaining neuronal health. Therapies for HD that reduce wild-type HTT may therefore generate unintended negative consequences
HD is an autosomal dominant neurodegenerative disorder caused by CAG-expansion in the huntingtin gene (HTT) that results in a toxic gain of function in the mutant huntingtin protein
potent and selective allele-specific knockdown of the mHTT protein can be achieved at therapeutically relevant SNP sites using ASOs in vitro and in vivo
Modification of these ASOs with S-constrained-ethyl (cET) motifs significantly improves potency while maintaining allele selectively in vitro
CAG-expansion in the huntingtin gene (HTT) that results in a toxic gain of function in the mutant huntingtin protein (mHTT)
