In vivo gene repair to restore healthy hematopoiesis in Fanconi anemia
Official title: In vivo interhomolog repair and selection of HSC in Fanconi Anemia
Funded at Children's Hosp of Philadelphia · NIH-11422867 · Data updated 2026-11-12
A study aims to repair faulty genes inside patients’ blood-forming cells to help people with Fanconi anemia regain healthy blood cell production.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | September 1, 2026 |
| Project end | May 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $452,150 |
| NIH project number | 1P01HL187309-01 |
| Project ID | NIH-11422867 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with Fanconi anemia or related bone marrow failure conditions who could benefit from novel gene repair strategies.
✕Less likely to help
Patients without Fanconi anemia or those whose disease is not driven by the targeted genetic mechanisms may not benefit.
What this research studies
This grant describes a new approach to fix defective genes directly inside patients’ hematopoietic stem cells using Cas9 nickase and lipid nanoparticles, with the goal of restoring a normal gene and improving blood cell formation. The work focuses on Fanconi anemia, a genetic disorder that causes bone marrow failure and cancer risk, and seeks to enable functional blood cells without needing donor transplant. The team plans to test interhomolog repair strategies in vivo, aiming for higher efficiency and fewer off-target effects. If successful, this could reduce treatment risks and broaden options for patients who have limited donor availability.
Why it matters
Potential benefit: If successful, patients with Fanconi anemia may experience improved blood cell production and fewer treatment-related risks.
How similar studies have gone: Earlier ex vivo gene therapies showed promise but faced challenges with stem cell numbers and long-term engraftment; this project explores a novel in vivo approach.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Kurre, Peter — Children's Hosp of Philadelphia
- Study coordinator: Kurre, Peter
Related research
Other NIH-funded projects on the same topics.
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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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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.