… oligonucleotides can be used to target either transcripts or duplex DNA and in diseases with
recessive inheritance oligonucleotides … , and novel strategies targeting DNA, instead of RNA…

… in disease and the prospects of targeting … diseases with ASOs is to target the expanded
CAG repeat allele with chemically modified CTG ASOs antisense to the CAG repeats of HTT or …

… Of the many ways to target the expression of RNA, this … oligonucleotides (ASOs) for therapy
of neurologic diseases. … CAG repeat expansion encoding a polyglutamine (polyQ) repeat, …

… (CUG)n triplet-repeat antisense oligonucleotides to effectively reduce mutant huntingtin …
Huntington's disease fibroblasts and lymphoblasts. The most effective antisense oligonucleotide, …

… However, because the mutation that causes HD is the expansion of a normally occurring
genetic element, selectively targeting muHTT with oligonucleotides presents significant …

… Depending on the chemistry and target site, antisense oligonucleotides can be used in …
Furthermore, one of the first repeat expansion diseases where antisense oligonucleotide …

… an antisense oligonucleotide (ASO) that is unique to the target … 13-mer DNA oligonucleotide
(oligo) targeting Rous sarcoma … CAG repeats are common in the genome and targeting the …

… : the discovery of biomarkers of disease progression and target engagement, advances
in … messenger RNA (mRNA) with antisense oligonucleotides (ASO) or RNA interference (RNAi)…

… under investigation for targeting RNA expression are antisense oligonucleotides (ASOs), …
disease, allele-specific strategies have been used: (1) by targeting the expanded CAG repeats …

… strategy is targeting the causative gene expression with oligonucleotide (ON) … targeting
SNP variants associated with mutations or targeting the pathologically expanded CAG repeat …