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EHDN 2022 | Huntingtin lowering: what level of wildtype protein knockdown is safe?

Michael Hayden, MB, ChB, PhD, FRCP, FRSC, The University of British Columbia, Vancouver, Canada, & Prilenia, CEO, discusses the unanswered research questions regarding the importance of wildtype huntingtin (HTT) and how this affects efforts to lower HTT therapeutically in Huntington’s disease (HD). While HTT is implicated in a large number of processes and interacts with many other proteins, more information is needed on its physiological function and the mechanisms by which polyglutamine tract expansion interferes with these roles to cause disease and degeneration. Given the clear role of mutant HTT in HD pathogenesis, HTT lowering has been a predominant investigational strategy for a number of years. Both agents that specifically target the mutant protein (allele specific) and those that unselectively target HTT (allele non-specific) have been studied. While some level of wildtype HTT knockdown appears to be safe, the amount that must be retained is unclear. Prof. Hayden comments on the importance of determining the level of knockdown that is both safe and effective when using non-selective approaches. This interview took place during the European Huntington’s Disease Network 2022 Plenary Meeting.

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