Understanding how ribosome production affects blood stem cells
Regulation of Ribosome Biogenesis in Hematopoietic Stem Cells
['FUNDING_R01'] · CHILDREN'S HOSP OF PHILADELPHIA · NIH-10911926
This study is looking at how certain proteins help blood-making stem cells work properly, especially in people with conditions like Shwachman-Diamond Syndrome, to find new ways to improve blood cell production for those who need it.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | CHILDREN'S HOSP OF PHILADELPHIA (nih funded) |
| Locations | 1 site (PHILADELPHIA, UNITED STATES) |
| Trial ID | NIH-10911926 on ClinicalTrials.gov |
What this research studies
This research investigates the role of ribosome biogenesis in the maintenance and function of hematopoietic stem cells (HSCs), which are crucial for producing blood cells. It focuses on how mutations in ribosomal proteins can lead to bone marrow failure syndromes, such as Shwachman-Diamond Syndrome, which can cause severe blood disorders. The study explores the mechanisms by which a specific protein, HectD1, regulates ribosome assembly and HSC function, aiming to uncover potential therapeutic targets for improving blood cell production in affected patients.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals diagnosed with bone marrow failure syndromes, particularly those with Shwachman-Diamond Syndrome or similar conditions.
Not a fit: Patients with bone marrow disorders not related to ribosome biogenesis or those without hematopoietic stem cell dysfunction may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatments for patients with bone marrow failure syndromes, improving their blood cell production and overall health.
How similar studies have performed: Previous research has shown promising results in understanding ribosome biogenesis and its implications for blood disorders, suggesting that this approach could yield valuable insights.
Where this research is happening
PHILADELPHIA, UNITED STATES
- CHILDREN'S HOSP OF PHILADELPHIA — PHILADELPHIA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: TONG, WEI — CHILDREN'S HOSP OF PHILADELPHIA
- Study coordinator: TONG, WEI
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.