Huntington's disease (HD) is an autosomal dominant inheritable neurodegenerative
disorder currently without effective treatment. It is caused by an expanded polyglutamine
(poly Q) tract in the corresponding protein, huntingtin (htt), and therefore suppressing the
huntingtin expression in brain neurons is expected to delay the onset and mitigate the
severity of the disease. Here, we have used small interfering RNAs (siRNAs) directed
against the huntingtin gene to repress the transgenic mutant huntingtin expression in an HD …