A Novel Tissue Engineering Technique to Repair Degenerated Retina
Grant Award Details
Grant Type:
Grant Number:
DISC1-09912
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Cell Line Generation:
Award Value:
$215,133
Status:
Closed
Progress Reports
Reporting Period:
Year 1
Grant Application Details
Application Title:
A Novel Tissue Engineering Technique to Repair Degenerated Retina
Public Abstract:
Research Objective
Transplantation of human embryonic stem cell (hESC) derived retina organoids (hESC-RO) together with hESC derived retinal pigment epithelium (hESC-RPE) to treat advanced retinal degeneration diseases
Impact
Based on the ‘proof of concept’ experiments in animal models, this novel approach can be translated into a therapeutic product for the treatment of advanced human retinal degenerative diseases.
Major Proposed Activities
Transplantation of human embryonic stem cell (hESC) derived retina organoids (hESC-RO) together with hESC derived retinal pigment epithelium (hESC-RPE) to treat advanced retinal degeneration diseases
Impact
Based on the ‘proof of concept’ experiments in animal models, this novel approach can be translated into a therapeutic product for the treatment of advanced human retinal degenerative diseases.
Major Proposed Activities
- Prepare hESC-RPE implants by culturing hESC-derived RPE cells (from H9 cells) on ultrathin parylene. hESC-RO’s will be derived from H9 cells (primarily based on the protocol of Zhong et al., 2014)
- A composite graft will be made of hESC-RPE and hESC-RO. A suitable surgical approach will be developed for subretinal placement of the co-graft in rats
- Assessment of visual function in transplanted rats by visual behavioral (optokinetic testing) and luminance threshold mapping of the superior colliculus (electrophysiological recording)
- Conduct morphological assessments of the tissue samples based on immunostaining and confocal microscopic imaging
Statement of Benefit to California:
The proposed co-graft approach will lead to the discovery of a new treatment strategy for retinal degeneration diseases such as age-related macular degeneration (AMD) and retinitis pigmentosa (RP). This approach will be beneficial for late stage disease conditions that are considered to be incurable because it requires replacement of both RPE and photoreceptors. By demonstrating the ‘proof of concept’ in animal disease models, it is easy to translate our findings to human clinical trials.