Lab-grown thymus cells to help T cell problems in CHARGE syndrome
Modeling and treating T cell immunodeficiency in CHARGE syndrome by ESC- and iPSC-derived thymic epithelial cells
Researchers are growing thymus-supporting cells from stem cells to help people with CHARGE syndrome who have low T cell counts.
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 ID | NIH-11283956 on NIH RePORTER |
What this research studies
If you or your child has CHARGE syndrome, this project uses embryonic and patient-derived induced pluripotent stem cells to create thymic epithelial cells in the lab so scientists can see how CHD7 genetic changes harm thymus development. The team will compare cells with and without CHD7 defects, attempt to correct or bypass those defects, and test whether the lab-made cells can form normal thymus tissue and support T cell growth. They will transplant these cells into animal models to see if they recreate thymic architecture and restore immune function. The goal is to learn whether this approach could become a future way to restore immunity for people with CHARGE-related T cell deficiency.
Who could benefit from this research
Good fit: Ideal candidates would be individuals with genetically confirmed CHARGE syndrome and evidence of T cell immunodeficiency, or those willing to provide cells or clinical data for research.
Not a fit: People without CHARGE syndrome or whose immune problems come from causes unrelated to thymic development are unlikely to benefit directly from this project.
Why it matters
Potential benefit: If successful, this work could point toward cell-based therapies that restore thymus function and improve T-cell immunity in people with CHARGE syndrome.
How similar studies have performed: Previous preclinical work has shown stem cell–derived thymic epithelial progenitors can form thymus-like tissue and support T cell development in mice, but applying this specifically to CHD7-related CHARGE syndrome is new.
Where this research is happening
Storrs-Mansfield, United States
- University of Connecticut Storrs — Storrs-Mansfield, United States (Active)
Researchers
- Principal investigator: Lai, Laijun — University of Connecticut Storrs
- Study coordinator: Lai, Laijun
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 below.