Investigating how a specific protein process affects beta-thalassemia
Official title: ULK-mediated autophagy of α-globin in ß-thalassemia
Funded at St. Jude Children's Research Hospital · NIH-10888292 · Data updated 2026-11-12
This study is looking at how a special protein can help reduce harmful buildup in the blood of people with beta-thalassemia, a condition that affects hemoglobin, and it aims to find new treatment options for patients by testing this in mice.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | St. Jude Children's Research Hospital NIH-funded |
| Lab location | 1 site (Memphis, United States) |
| Project start | July 1, 2022 |
| Project end | June 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $639,532 |
| NIH project number | 5R01HL165798-03 |
| Project ID | NIH-10888292 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with beta-thalassemia, particularly those experiencing severe symptoms or complications.
✕Less likely to help
Patients with other forms of anemia or blood disorders unrelated to beta-thalassemia may not benefit from this research.
What this research studies
This research focuses on understanding the role of protein quality control in beta-thalassemia, a blood disorder caused by mutations that affect hemoglobin production. It explores how the Unc-51–like autophagy activating kinase 1 (ULK1) can help manage the toxic buildup of free alpha-globin, which is harmful to red blood cell development. By using a preclinical mouse model, the study examines the effects of inhibiting a specific kinase to promote the autophagy process, potentially leading to new treatment strategies for patients suffering from this condition.
Why it matters
Potential benefit: If successful, this research could lead to innovative therapies that improve the management of beta-thalassemia and reduce complications associated with the disease.
How similar studies have gone: Previous research has shown promise in using autophagy modulation for similar blood disorders, indicating potential for success in this novel approach.
Where this research is happening
-
St. Jude Children's Research Hospital
Memphis, United States Active
Researchers
- Principal investigator: Weiss, Mitchell J — St. Jude Children's Research Hospital
- Study coordinator: Weiss, Mitchell J
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Clearing excess alpha-globin to help people with beta-thalassemia
This research looks at whether boosting a cell 'self-cleaning' pathway (ULK1-driven autophagy) can remove toxic excess alpha-globin and help people with beta-thalassemia.
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.