RNA-directed therapy for Huntington’s disease

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Grant Award Details

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Human Stem Cell Use:
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Progress Reports

Reporting Period:
Year 2

Grant Application Details

Application Title:

RNA-directed therapy for Huntington's disease

Public Abstract:
Research Objective

We develop a novel adeno-associated viral (AAV) vector-delivered RNA-targeting therapeutic for elimination of toxic RNA causative of Huntington’s disease.


There are no disease-modifying therapies for Huntington’s disease. Our therapeutic, if successful, will be a first-in-class treatment for this invariably fatal neurodegenerative disorder.

Major Proposed Activities

  • In vitro studies of the RNA-targeting system in human Huntington's disease patient stem cell derived striatal organoids to assess the ability to eliminate toxic RNA foci
  • AAV vector packaging of the CAG-targeting RNA-targeting system to obtain high-titer viral preparations, and in vivo (mouse) safety studies to assess immunogenicity, cytotoxicity and off-target effects
  • In vivo efficacy studies of the RNA-targeting system in a mouse model of Huntington's disease to assess effects on disease-relevant molecular, cellular, behavioral and motor function deficits
Statement of Benefit to California:
Currently, there is no cure for Huntington’s disease, which currently affects thousands of Californians. The California population will equitably benefit from the development of a therapeutic for Huntington’s disease, which affects the state's residents roughly equally across gender, race/ethnicity and socioeconomic status. Our therapeutic strategy is readily transferrable to a large set of other devastating diseases, multiplying the benefits of development of this new therapeutic modality.