Targeting and degrading mutant transcription factors to treat blood disorders
Official title: Development of selective degradation strategies towards mutant transcription factors
Funded at Icahn School of Medicine at Mount Sinai · NIH-11057325 · Data updated 2026-11-12
This study is exploring new ways to safely break down faulty proteins that can lead to blood disorders like congenital dyserythropoietic anemia, with the hope of finding better treatments for people dealing with these genetic issues.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Icahn School of Medicine at Mount Sinai NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | September 15, 2024 |
| Project end | August 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $253,500 |
| NIH project number | 1R21DK139543-01A1 |
| Project ID | NIH-11057325 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients diagnosed with congenital dyserythropoietic anemia or other blood disorders linked to mutations in transcription factors.
✕Less likely to help
Patients with blood disorders not related to transcription factor mutations or those with complex multi-allelic conditions may not benefit from this research.
What this research studies
This research focuses on developing innovative strategies to selectively degrade mutant transcription factors that can cause blood disorders such as congenital dyserythropoietic anemia. By introducing specific degradative modules that target the mutant variant of the KLF1 transcription factor, the research aims to restore normal gene expression patterns in affected cells. The approach includes both in vitro tests and in vivo cell culture experiments to evaluate the effectiveness of these targeted degradation methods. If successful, this could lead to new treatments for patients with similar genetic mutations affecting their blood health.
Why it matters
Potential benefit: If successful, this research could provide a novel therapeutic approach for patients suffering from blood disorders caused by specific genetic mutations.
How similar studies have gone: While the approach of selectively degrading mutant proteins is innovative, similar strategies have shown promise in other hematologic conditions, suggesting potential for success.
Where this research is happening
-
Icahn School of Medicine at Mount Sinai
New York, United States Active
Researchers
- Principal investigator: Bieker, James J — Icahn School of Medicine at Mount Sinai
- Study coordinator: Bieker, James J
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.