Article
mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo.
Nucleic acid therapeutics - 1 Aug 2024
Allen Sarah, O'Reilly Daniel, Miller Rachael, Sapp Ellen, Summers Ashley, Paquette Joseph, Echeverria Moreno Dimas, Bramato Brianna, McHugh Nicholas, Yamada Ken, Aronin Neil, DiFiglia Marian, Khvorova Anastasia
Abstract excerpt
Huntington's disease (HD) is an autosomal dominant neurodegenerative disease caused by CAG repeat expansion in the first exon of the huntingtin gene (HTT). Oligonucleotide therapeutics, such as short interfering RNA (siRNA), reduce levels of huntingtin mRNA and protein in vivo and are considered a viable therapeutic strategy. However, the extent to which they silence huntingtin mRNA in the nucleus is not...
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