2024
DOI: 10.3390/ijms252111493
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Regulation of HTT mRNA Biogenesis: The Norm and Pathology

Alexandra E. Zubkova,
Dmitry V. Yudkin

Abstract: Huntington’s disease (HD) is a neurodegenerative disorder caused by the expansion of the CAG repeat in exon 1 of the HTT gene, leading to the formation of a toxic variant of the huntingtin protein. It is a rare but severe hereditary disease for which no effective treatment method has been found yet. The primary therapeutic targets include the mutant protein and the mutant mRNA of HTT. Current clinical trial approaches in gene therapy involve the application of splice modulation, siRNA, or antisense oligonucleo… Show more

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