Drug discovery for Charcot Marie Tooth Disease using hPSC-derived Schwann cells
Grant Award Details
Grant Type:
Grant Number:
DISC2-16715
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$2,471,664
Status:
Active
Grant Application Details
Application Title:
Drug discovery for Charcot Marie Tooth Disease using hPSC-derived Schwann cells
Public Abstract:
Research Objective
The therapeutic candidate we aim to discover under this award is clomipramine, an antidepressant that might help reduce a harmful protein in the nerve cells of patients with CMT1A disease.
Impact
This therapeutic candidate has the potential to treat debilitating neurological symptoms in patients with CMT1A.
Major Proposed Activities
The therapeutic candidate we aim to discover under this award is clomipramine, an antidepressant that might help reduce a harmful protein in the nerve cells of patients with CMT1A disease.
Impact
This therapeutic candidate has the potential to treat debilitating neurological symptoms in patients with CMT1A.
Major Proposed Activities
- Initial assessment of clomipramine in human stem cell derived CMT1A myelin cells
- ExtendedCharacterization of clomipramine in human stem cell derived CMT1A nerve models
- Efficacy assessment in transplanted human stem cell derived CMT1A myelin cells
- Efficacy studies of clomipramine in mouse models of CMT1A
- Understanding the mechanism of action of clomipramine
- Data analysis and reporting of findings
Statement of Benefit to California:
The proposed research will benefit the State of California by potentially providing a new therapy for a debilitating neurological disorder that affects many patients. This advancement could improve their quality of life, reducing healthcare costs associated with long-term care and disability. Additionally, the research will bolster the state's position as a leader in biomedical innovation, creating opportunities for local biotech companies and fostering growth in the life sciences sector.
