Understanding and adjusting telomerase in blood cells
Regulation and manipulation of the telomerase RNA component in hematopoiesis
This project aims to understand how a key component called telomerase works in human blood cells to develop new treatments for various diseases.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-11085202 on NIH RePORTER |
What this research studies
Telomerase is vital for healthy cells, but we don't fully understand how it's controlled in people. This knowledge gap makes it hard to create treatments for conditions caused by telomerase problems. Our goal is to learn how to adjust telomerase in human cells, especially those in the blood system, to help patients. We are focusing on a specific part of telomerase called TERC, which, when low due to genetic changes, can lead to serious conditions like aplastic anemia and certain heart diseases. We are also looking at a protein called PAPD5, which seems to control TERC levels, and hope to find ways to block PAPD5 to restore healthy telomerase function.
Who could benefit from this research
Good fit: Patients with conditions linked to low telomerase RNA component (TERC) levels, such as dyskeratosis congenita, aplastic anemia, myelodysplastic syndromes, or certain cardiovascular diseases, could potentially benefit from future therapies developed from this research.
Not a fit: Patients whose conditions are not related to telomerase dysfunction or low TERC levels would likely not benefit from this specific therapeutic approach.
Why it matters
Potential benefit: If successful, this work could lead to new medications that restore healthy telomerase function, potentially treating or preventing a range of degenerative disorders, particularly those affecting blood cells.
How similar studies have performed: While the role of telomerase in disease is well-established, targeting PAPD5 to restore TERC levels is a novel approach that is currently being explored for its therapeutic effectiveness.
Where this research is happening
Boston, United States
- Boston Children's Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Agarwal, Suneet — Boston Children's Hospital
- Study coordinator: Agarwal, Suneet
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.