Investigating rare genetic variants linked to schizophrenia using engineered stem cells
Official title: Functional characterization of schizophrenia rare variants using genetically engineered human iPSCs
Funded at Johns Hopkins University · NIH-10984986 · Data updated 2026-11-12
This study is looking at how certain rare genetic changes might play a role in schizophrenia by creating special cells that mimic these changes, which could help us understand the condition better and find new ways to treat it.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Johns Hopkins University NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project start | January 1, 2023 |
| Project end | November 30, 2027 |
| Funding year | 2025 |
| Award (this year) | $594,993 |
| NIH project number | 5R01MH129277-03 |
| Project ID | NIH-10984986 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with a family history of schizophrenia or those who have been diagnosed with the disorder and have rare genetic variants.
✕Less likely to help
Patients without a genetic predisposition to schizophrenia or those with unrelated mental health conditions may not benefit from this research.
What this research studies
This research focuses on understanding how rare genetic variants contribute to schizophrenia by using genetically engineered human induced pluripotent stem cells (hiPSCs). The researchers will create specific cell lines that carry mutations associated with schizophrenia and observe how these mutations affect neuronal development and function. By employing advanced gene editing techniques, they aim to uncover the biological mechanisms that underlie the disorder, which could lead to new insights into treatment options. Patients may benefit from this research as it seeks to clarify the genetic factors involved in schizophrenia, potentially leading to more targeted therapies.
Why it matters
Potential benefit: If successful, this research could lead to improved understanding and treatment options for patients with schizophrenia.
How similar studies have gone: Other research has successfully utilized similar approaches with iPSCs to study genetic disorders, indicating a promising avenue for understanding schizophrenia.
Where this research is happening
-
Johns Hopkins University
Baltimore, United States Active
Researchers
- Principal investigator: Li, Pan — Johns Hopkins University
- Study coordinator: Li, Pan
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.