Antisense oligonucleotides (ASOs) have been investigated as a huntingtin (HTT) lowering therapeutic approach in Huntington’s disease. These ASOs are designed to bind specifically to HTT mRNA, triggering RNase H1-mediated degradation of the mRNA and consequential reductions in HTT protein concentration. Patrick Weydt, MD, PhD, University of Bonn Medical Center, Bonn, Germany, discusses the value of ASOs in the HD setting, compared to other gene therapy techniques. For example, adeno-associated virus (AAV) vector-based gene therapies in development are designed to change the DNA sequence through transgene introduction and microRNA production. These strategies are appealing as they only require a one-time administration. CRISPR-Cas9 mediated genome editing for HD is also a promising strategy under investigation. Dr Weydt notes that the non-permanent nature of ASOs may be beneficial in these early stages while investigations into safety and long-term outcomes are ongoing. This interview took place during the European Huntington’s Disease Network 2022 Plenary Meeting.
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