Global gene expression profiling of single inhibitory neurons derived from human stem cells
Grant Award Details
Grant Number:
DISC2P-11700
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$202,500
Status:
Closed
Progress Reports
Reporting Period:
Year 1
Grant Application Details
Application Title:
Global gene expression profiling of single inhibitory neurons derived from human stem cells
Public Abstract:
Research Objective
This study will use high throughput single-cell capture and next-generation sequencing technologies to resolve the composition of an emerging neuronal cell therapy product.
Impact
If successful, this study will facilitate robust manufacturing processes and analytical methods toward a potentially safer and more effective cell therapy product.
Major Proposed Activities
["Characterize the cellular composition of three independent lots of product", "Determine the long-term cell fate of the product following transplantation into a preclinical animal model"]
This study will use high throughput single-cell capture and next-generation sequencing technologies to resolve the composition of an emerging neuronal cell therapy product.
Impact
If successful, this study will facilitate robust manufacturing processes and analytical methods toward a potentially safer and more effective cell therapy product.
Major Proposed Activities
["Characterize the cellular composition of three independent lots of product", "Determine the long-term cell fate of the product following transplantation into a preclinical animal model"]
Statement of Benefit to California:
If successful, this study will directly benefit the 400,000 people living with epilepsy in the State of California, of which approximately one-third are multi-drug resistant. Additionally, the single-cell capture, sequencing, and gene expression analysis tools could potentially be used to improve and accelerate the development of other regenerative stem cell-based therapies.
Publications
- Cell Stem Cell (2023): Human pallial MGE-type GABAergic interneuron cell therapy for chronic focal epilepsy. (PubMed: 37802038)