Article
Allele-Selective Inhibition of Mutant Huntingtin with 2-Thio- and C5- Triazolylphenyl-Deoxythymidine-Modified Antisense Oligonucleotides.
Nucleic acid therapeutics - 1 Oct 2015
Østergaard Michael E, Kumar Pawan, Nichols Josh, Watt Andrew, Sharma Pawan K, Nielsen Poul, Seth Punit P
Abstract excerpt
We report the effect of introducing a single incorporation of 2-thio-deoxythymidine (2S-dT) or C5-Triazolylphenyl-deoxythymidine (5-TrPh-dT) at four positions within the gap region of RNase H gapmer antisense oligonucleotides (ASOs) for reducing wild-type and mutant huntingtin mRNA in human patient fibroblasts. We show that these modifications can modulate processing of the ASO/RNA heteroduplexes by recombinant...
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