Gene Silencing Therapies for Huntington’s Disease
Huntington’s disease (HD) is a rare, autosomal‑dominant neurodegenerative disorder precipitated by a pathological expansion of CAG trinucleotide repeats in exon 1 of the huntingtin (HTT) gene. Mutant huntingtin (mHTT) imposes a toxic gain‑of‑function that progressively compromises neuronal circuitry...
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| Main Authors: | , |
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| Format: | Article |
| Language: | English |
| Published: |
Iran University of Medical Sciences
2025-05-01
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| Series: | Neurology Letters |
| Subjects: | |
| Online Access: | https://www.neurologyletters.com/article_220437_bd85211e84319a406a0d5d56fd626a43.pdf |
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