Article

mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs <i>in vivo</i>

2024-04-28

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 HTT mRNA in the nucleus is not established....

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Literature Corpus work: 63472fd3-11d2-50e9-99e3-a52e9e0466ef
DOI: 10.1101/2024.04.24.590997

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