Helping blood-forming stem cells grow better in the lab for stronger transplants and gene therapies
Modulation of Endoplasmic Reticulum Associated Degradation to Support Ex Vivo Expansion of Hematopoietic Stem Cells
This project tries ways to protect and expand blood-forming stem cells grown in the lab so they can work better for people needing bone marrow transplants or gene therapy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Michigan at Ann Arbor NIH-funded |
| Lab location | 1 site (Ann Arbor, United States) |
| Project ID | NIH-11319883 on NIH RePORTER |
What this research studies
The team studies long-term hematopoietic stem cells (the blood-forming stem cells used for transplants) and how protein quality control inside the cell, called ER-associated degradation (ERAD), affects their survival when grown outside the body. They will manipulate ERAD pathways in lab cultures and use molecular tests to preserve stem cell function during ex vivo expansion. The work uses human-relevant cells and lab models to try to produce more transplantable, gene-modified stem cells without exhausting their ability to rebuild the blood system. Successful lab findings would guide further preclinical work and possible future clinical testing.
Who could benefit from this research
Good fit: This work is most relevant to people who need hematopoietic stem cell transplantation or gene therapy for blood disorders such as leukemia, sickle cell disease, inherited immunodeficiencies, or other bone marrow failure syndromes.
Not a fit: People with conditions unrelated to the blood or bone marrow, or those needing immediate treatment, are unlikely to benefit directly from this lab-focused work.
Why it matters
Potential benefit: If successful, this could make it easier to produce enough healthy, functional stem cells so more patients can receive curative transplants or gene therapies.
How similar studies have performed: Past laboratory efforts to expand human HSCs have shown limited success, and targeting ERAD is a newer approach with promising early data but not yet proven in patients.
Where this research is happening
Ann Arbor, United States
- University of Michigan at Ann Arbor — Ann Arbor, United States (Active)
Researchers
- Principal investigator: Li, Qing — University of Michigan at Ann Arbor
- Study coordinator: Li, Qing
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.