New methods for gene therapy and bone marrow transplants in Fanconi anemia
Official title: Nongenotoxic conditioning for gene therapy and allogeneic transplantation in Fanconi anemia
Funded at Fred Hutchinson Cancer Center · NIH-10656502 · Data updated 2026-11-12
This study is looking for better ways to help people with Fanconi anemia by using safer treatments that avoid harsh chemotherapy and radiation before they get a bone marrow or stem cell transplant, making the process easier and reducing the chance of future cancers.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Fred Hutchinson Cancer Center NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project start | December 20, 2019 |
| Project end | November 30, 2025 |
| Funding year | 2023 |
| Award (this year) | $811,794 |
| NIH project number | 5R01HL147324-05 |
| Project ID | NIH-10656502 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Fanconi anemia who require a bone marrow or stem cell transplant.
✕Less likely to help
Patients with Fanconi anemia who are not candidates for transplantation or those with advanced cancer may not benefit from this research.
What this research studies
This research focuses on improving treatments for Fanconi anemia, a genetic disorder that leads to bone marrow failure and increased cancer risk. The team is exploring nongenotoxic conditioning methods, which avoid harmful chemotherapy and radiation, to prepare patients for bone marrow or stem cell transplants. By using antibody drug conjugates that target specific stem cells, the goal is to enhance the success of transplants while minimizing the risk of secondary cancers. This innovative approach aims to provide safer and more effective treatment options for patients with this condition.
Why it matters
Potential benefit: If successful, this research could lead to safer bone marrow transplants and improved outcomes for patients with Fanconi anemia.
How similar studies have gone: Previous research has shown promise in using antibody drug conjugates for similar applications, indicating potential for success in this novel approach.
Where this research is happening
-
Fred Hutchinson Cancer Center
Seattle, United States Active
Researchers
- Principal investigator: Kiem, Hans-Peter — Fred Hutchinson Cancer Center
- Study coordinator: Kiem, Hans-Peter
Related research
Other NIH-funded projects on the same topics.
-
In vivo gene repair to restore healthy hematopoiesis in Fanconi anemia
A study aims to repair faulty genes inside patients’ blood-forming cells to help people with Fanconi anemia regain healthy blood cell production.
-
Understanding genetic dependencies when Fanconi anemia pathway is lost
A study to find other genes or pathways that cancer cells rely on when the Fanconi anemia repair system is broken, with the aim of guiding future treatments.
-
Innovative cell and gene therapy approaches to treat Fanconi anemia
A Stanford-led project aims to develop safer cell and gene therapies to help people with Fanconi anemia who have bone marrow failure.
-
How cells detect DNA damage and activate ATR, ATM, and Fanconi anemia repair systems
Researchers are using high-resolution structural imaging to show how key repair proteins sense DNA damage, with relevance for people whose cancers come from DNA repair defects.
-
In vivo gene therapy for Fanconi anemia
A patient-focused overview of a potential in vivo gene therapy approach to correct Fanconi anemia, aiming to reduce the need for donor transplants.
-
Fanconi anemia: what happens inside cells
Researchers want to learn how proteins called REV7 and CHAMP1 change DNA repair in people with Fanconi anemia and related genetic syndromes.
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.