Correcting FANCC mutations for treating Fanconi Anemia
FANCC mutation correction using homology-independent targeted integration for gene therapy of Fanconi Anemia group C
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 ID | NIH-10886717 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Fanconi Anemia, particularly those with mutations in the FANCC gene.
Not a fit: Patients with Fanconi Anemia caused by mutations in genes other than FANCC may not benefit from this specific research.
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 performed: 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
Indianapolis, United States
- Indiana University Indianapolis — Indianapolis, United States (Active)
Researchers
- Principal investigator: Tran, Ngoc Tung — Indiana University Indianapolis
- Study coordinator: Tran, Ngoc Tung
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 below.