In vivo gene therapy for Fanconi anemia
Funded at Fred Hutchinson Cancer Center · NIH-11297452 · Data updated 2026-11-12
A patient-focused overview of a potential in vivo gene therapy approach to correct Fanconi anemia, aiming to reduce the need for donor transplants.
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 | March 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $835,731 |
| NIH project number | 2R01HL147324-06A1 |
| Project ID | NIH-11297452 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and adolescents with Fanconi anemia who lack an HLA-matched donor and are candidates for gene therapy approaches.
✕Less likely to help
Patients with conditions outside Fanconi anemia or those not eligible for the specific in vivo therapy studied.
What this research studies
Fanconi anemia is a serious inherited bone marrow failure condition where finding a matching donor for transplantation is often difficult and risky. This grant explores delivering a functional gene directly inside the patient’s body to repair blood-forming cells, potentially reducing reliance on complex stem cell transplants. The research will compare two promising in vivo gene therapy strategies, focusing on safety, efficiency, and the ability to restore healthy blood cell production. If successful, this could lead to a faster, less toxic treatment option for children and young people with FA.
Why it matters
Potential benefit: If successful, this approach could offer a safer, less burdensome treatment for Fanconi anemia by repairing blood-forming cells inside the body.
How similar studies have gone: Prior ex vivo gene therapy studies showed partial improvements but faced challenges; in vivo approaches are promising but still early.
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.
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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.
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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.
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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.
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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.
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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.
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Understanding how cells detect and respond to DNA damage
This study is looking at how our cells detect and fix damage to their DNA, which is really important for stopping cancer, and it aims to understand how certain proteins work together to repair serious DNA problems, with the hope that this knowledge can help develop new treatments to improve DNA repair in cancer cells.
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.