Grant Award Details

RNA-directed therapy for Huntington's disease
Grant Number: 
DISC2-13102
Project Objective: 
  • To develop an AAV-delivered transgene targeting CAG repeats in Huntington Disease
Investigator: 
Name: 
Type: 
PI
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.