Correcting FANCC mutations for treating Fanconi Anemia
Official title: FANCC mutation correction using homology-independent targeted integration for gene therapy of Fanconi Anemia group C
Funded at Indiana University Indianapolis · NIH-10886717 · Data updated 2026-11-12
This study is testing a new gene therapy that uses cutting-edge technology to fix a problem in the FANCC gene for people with Fanconi Anemia, aiming to help their blood stem cells work better and provide a safer treatment option than traditional stem cell transplants.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Indiana University Indianapolis NIH-funded |
| Lab location | 1 site (Indianapolis, United States) |
| Project start | July 15, 2023 |
| Project end | June 30, 2025 |
| Funding year | 2024 |
| Award (this year) | $237,750 |
| NIH project number | 5R21HL168669-02 |
| Project ID | NIH-10886717 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Fanconi Anemia, particularly those with mutations in the FANCC gene.
✕Less likely to help
Patients with Fanconi Anemia caused by mutations in genes other than FANCC may not benefit from this specific research.
What this research studies
This research focuses on developing a gene therapy approach to correct mutations in the FANCC gene, which is responsible for Fanconi Anemia, a serious inherited disorder. Using advanced CRISPR/Cas9 technology, the researchers aim to modify the genome of blood stem cells from affected patients, allowing for the potential restoration of normal function. The corrected stem cells would then be transplanted back into the patient, offering a new treatment option that avoids the complications associated with traditional stem cell transplants. This innovative method seeks to provide a safer and more effective solution for managing the disease.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients with Fanconi Anemia, potentially improving their quality of life and survival rates.
How similar studies have gone: Previous research using CRISPR technology for gene correction in blood disorders has shown promising results, indicating potential success for this approach.
Where this research is happening
-
Indiana University Indianapolis
Indianapolis, United States Active
Researchers
- Principal investigator: Tran, Ngoc Tung — Indiana University Indianapolis
- Study coordinator: Tran, Ngoc Tung
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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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.
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.