Name:
Institution:
Type:
PI
Grant Award Details
RNA-directed therapy for Huntington's disease
Grant Type:
Grant Number:
DISC2-13102
Project Objective:
- To develop an AAV-delivered transgene targeting CAG repeats in Huntington Disease
Investigator:
Disease Focus:
Huntington's Disease
Neurological Disorders
Human Stem Cell Use:
iPS Cell
Award Value:
$1,253,110
Status:
Active
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.
Impact
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.
