New study highlights challenges in targeting mutant huntingtin mRNA in Huntington’s disease.
- A study on Huntington’s disease found that short interfering RNA (siRNA) is ineffective in reducing mutant huntingtin gene mRNA in the nucleus of mouse brains.
- Mutant mRNA forms nuclear clusters that are resistant to RNA interference (RNAi), while wild-type mRNA in both the nucleus and cytoplasm can be silenced.
- This study highlights the impact of the RNA structure in the nucleus on the efficiency of RNAi-based silencing.
- The research provides new understanding of the relationship between huntingtin RNA and protein levels and their functional implications.
- The study is published in the journal Nucleic Acid Therapeutics and contributes valuable information to the field of Huntington’s disease research.
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Neurology, Genetics, RNAi, Huntingtin, Gene Therapy