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A5 RNA targeted therapies, mechanisms, and the challenges of delivery in HD
  1. Douglas Macdonald
  1. CHDI Foundation/CHDI Management, Los Angeles, CA, USA

Abstract

Huntingtin protein (HTT) lowering is a key therapeutic strategy for Huntington’s disease (HD). Reducing the amount of the disease-causing expanded HTT protein in the brain of patients is predicted to slow the progression of the disease. Several approaches are being employed to lower HTT including antisense oligonucleotides and siRNAs, as well as gene therapy approaches using viral delivery of miRNAs, shRNAs, and zinc-finger repressor proteins. Each of these potential therapeutic agents has inherent distribution challenges requiring direct delivery to the CNS. These challenges will be discussed as well as the pharmacodynamic biomarkers aimed to validate that delivery of a HTT lowering therapy does, in fact, lower the amount of HTT protein in the brains of HD patients.

  • HTT lowering
  • HTT protein
  • biomarker

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