Grant Award Details
- To develop an AAV-delivered transgene targeting CAG repeats in Huntington Disease
Grant Application Details
- RNA-directed therapy for Huntington's disease
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
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.