Abstract
Huntington disease (HD) is an autosomal dominant inherited neurodegenerative disease that is caused by a gain of function mutation due to the expansion of an unstable CAG trinucleotide repeat in exon 1 of the huntingtin (htt) gene. Using an inducible mouse model of HD it has previously been demonstrated that abolishment of the expression of the mutant protein not only halted progression of the disease but also reversed aggregate formation and neuronal degeneration. Therefore, blocking the expression of mutant htt might be a promising therapeutic strategy for HD. Small interference RNA (siRNA) has been shown to be a potent tool to inhibit specific gene expression in vitro and more recently also in vivo. We elected to adapt the siRNA technology for adenovirus-mediated inhibition of htt expression as a new treatment strategy for HD.
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