Development of an Optogenetic Vision Restoration Gene Therapy Using an Engineered Form of Melanopsin
Grant Award Details
Grant Type:
Grant Number:
DISC2-14963
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$1,150,820
Status:
Active
Grant Application Details
Application Title:
Development of an Optogenetic Vision Restoration Gene Therapy Using an Engineered Form of Melanopsin
Public Abstract:
Research Objective
The objective of this research proposal is to develop a lead AAV candidate for an optogenetic vision restoration therapy for patients suffering from blindness due to loss of photoreceptors.
Impact
Our optogenetic vision restoration AAV gene therapeutic candidate would non-invasively restore sight to patients terminally blinded by photoreceptors loss.
Major Proposed Activities
The objective of this research proposal is to develop a lead AAV candidate for an optogenetic vision restoration therapy for patients suffering from blindness due to loss of photoreceptors.
Impact
Our optogenetic vision restoration AAV gene therapeutic candidate would non-invasively restore sight to patients terminally blinded by photoreceptors loss.
Major Proposed Activities
- Complete Retinal Explant Characterization of Optogenetic Candidates and Select Leads
- Complete Development of an Optogenetic Retinal AAV Expression Cassette (pOR)
- Generate & Evaluate AAV Test Candidates for Animal Studies
- Visual Rescue of Blind Rd1 Mice with AAV Test Candidates
- Complete Tropism, Biodistribution, and Tolerability Evaluation of Lead AAV in NHP
Statement of Benefit to California:
Blindness imposes both a personal burden on patients as well as a financial burden upon the State of California due to costs related to care and loss of productivity. With the number of blind people set to increase due to aging demographics and increased prevalence of blinding diseases such as DryAMD, a therapeutic to restore vision would provide relief to both Californian patients & their families personally and as well as the State through reduced care costs and increased productivity.