Innovative cell and gene therapy approaches to treat Fanconi anemia
Official title: Developing Innovative Cell and Gene Therapy Strategies to Treat Fanconi Anemia
Funded at Stanford University · NIH-11416385 · Data updated 2026-11-12
A Stanford-led project aims to develop safer cell and gene therapies to help people with Fanconi anemia who have bone marrow failure.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Stanford University NIH-funded |
| Lab location | 1 site (Stanford, United States) |
| Project start | September 15, 2025 |
| Project end | June 30, 2030 |
| Funding year | 2026 |
| Award (this year) | $661,057 |
| NIH project number | 5R01HL179280-02 |
| Project ID | NIH-11416385 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with Fanconi anemia or related bone marrow failure conditions who may be candidates for future cell or gene therapy approaches.
✕Less likely to help
Patients not eligible for gene- or cell-based therapies or those with conditions outside the FA spectrum may not benefit.
What this research studies
This grant focuses on creating non-toxic cell and gene therapy options for Fanconi anemia, aiming to replace harsh conditioning used before stem cell transplantation. Researchers are testing strategies that allow functional blood-forming cells to take root without damaging DNA. The work includes using antibodies and antibody-drug conjugates to prepare the body for new stem cells and exploring autologous gene therapy and mosaic approaches. The ultimate goal is to enable durable blood cell production with fewer treatment-related risks for FA patients.
Why it matters
Potential benefit: If successful, patients with Fanconi anemia could receive effective treatments with fewer side effects and better long-term blood cell function.
How similar studies have gone: Previous work shows that small numbers of functional HSPCs can stabilize hematopoiesis in FA, suggesting potential for these approaches.
Where this research is happening
-
Stanford University
Stanford, United States Active
Researchers
- Principal investigator: Czechowicz, Agnieszka — Stanford University
- Study coordinator: Czechowicz, Agnieszka
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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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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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.
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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.