Fortschritte der Neurologie-Psychiatrie
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Fortschr Neurol Psychiatr · Aug 2017
Review[Hope for Huntington's disease patients: first clinical gene silencing study in progress].
Huntington's disease (HD) is an autosomal-dominant inherited neurodegenerative disorder, characterized by motor, psychiatric and cognitive symptoms for which as yet no causal treatment is available. It has a prevalence of 1 : 10 000 in Germany. Its cause is a mutation in the Huntington gene (CAG-repeat). ⋯ One way to achieve gene silencing is the use of antisense oligonucleotides (ASO) that bind to pre-mRNA. Since August 2015, a first clinical trial with ASO (study drug: IONIS-HTTRx) in early manifest HD patients is in progress (NCT02519036). Results from this study could lead to a first causal treatment option in HD.