Understanding and treating T cell issues in CHARGE syndrome using specialized cells
Official title: Modeling and treating T cell immunodeficiency in CHARGE syndrome by ESC- and iPSC-derived thymic epithelial cells
Funded at University of Connecticut Storrs · NIH-11009900 · Data updated 2026-11-12
This study is looking at how special cells made from stem cells can help people with CHARGE syndrome develop healthier immune systems by improving the function of their thymus, which is important for making T cells.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Connecticut Storrs NIH-funded |
| Lab location | 1 site (Storrs-Mansfield, United States) |
| Project start | February 1, 2023 |
| Project end | January 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $438,967 |
| NIH project number | 5R01AI175087-03 |
| Project ID | NIH-11009900 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with CHARGE syndrome who experience T cell immunodeficiency.
✕Less likely to help
Patients without CHARGE syndrome or those whose immunodeficiency is not related to thymic development may not benefit from this research.
What this research studies
This research focuses on CHARGE syndrome, a condition that can lead to significant T cell immunodeficiency due to problems with thymus development. The study aims to investigate how thymic epithelial cells, derived from embryonic stem cells, can be used to restore normal thymic function and support T cell development in affected individuals. By using advanced cell differentiation techniques, researchers hope to create a supportive environment for T cell maturation, which is crucial for a healthy immune response. Patients with CHARGE syndrome may benefit from this innovative approach to improve their immune function.
Why it matters
Potential benefit: If successful, this research could significantly enhance immune function in patients with CHARGE syndrome, potentially reducing their susceptibility to infections.
How similar studies have gone: While this approach is innovative, similar strategies using stem cell-derived thymic epithelial cells have shown promise in preliminary studies, indicating potential for success.
Where this research is happening
-
University of Connecticut Storrs
Storrs-Mansfield, United States Active
Researchers
- Principal investigator: Lai, Laijun — University of Connecticut Storrs
- Study coordinator: Lai, Laijun
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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.