Identifying small molecules that stimulate the differentiation of hESCs into dopamine-producing neurons
Identifying small molecules that stimulate the differentiation of hESCs into dopamine-producing neurons
Funding Type:
SEED Grant
Grant Number:
RS1-00215
Investigator:
Funds Committed:
$564,309
Disease Focus:
Parkinson's Disease
Stem Cell Use:
Embryonic Stem Cell
Status:
Closed
Public Abstract:
In this application, we propose to identify small molecule compounds that can stimulate human embryonic stem cells to become dopamine-producing neurons. These neurons degenerate in Parkinson’s disease, and currently have very limited availability, thus hindering the cell replacement therapy for treating Parkinson’s disease.
Our proposed research, if successful, will lead to the identification of small molecule compounds that can not only stimulate cultured human embryonic stem cells to become DA neurons, but may also stimulate endogenous brain stem cells to regenerate, since the small molecule compounds can be made readily available to the brain due to their ability to cross the blood-brain barrier. In addition, these small molecule compounds may serve as important research tools, which can tell us the fundamental biology of the human embryonic stem cells.
Statement of Benefit to California:
The proposed research will potentially lead to a cure for the devastating neurodegenerative, movement disorder, Parkinson’s disease. The proposed research will potentially provide important research tools to better understand hESCs. Such improved understanding of hESCs may lead to better treatments for a variety of diseases, in which a stem-cell based therapy could make a difference.
Progress Report:
Year 1
Parkinson’s disease is the most common movement disorder due to the degeneration of brain dopaminergic neurons. One strategy to combat the disease is to replenish these neurons in the patients, either through transplantation of stem cell-derived dopaminergic neurons, or through promoting endogenous dopaminergic neuronal production or survival. We have carried out a small molecule based screen to identify compounds that can affect the development and survival of dopaminergic neurons from pluripotent stem cells. The small molecules that we have identified will not only serve as important research tools for understanding dopaminergic neuron development and survival, but potentially could also lead to therapeutics in the induction of dopaminergic neurons for treating Parkinson’s disease.Current RFAs
CIRM funds grants through directed Requests for Applications (RFAs).
Workshop Reports
CIRM holds regular scientific workshops and meetings to update the scientific staff and CIRM grantees about particular areas of stem cell and related research.
Cerebral Palsy Workshop Report [pdf]
Human iPS Cell Banking Workshop [pdf]
SCNT Workshop Report [pdf]
