Understanding how neurofibromin affects cancer growth in patients with Neurofibromatosis Type 1.
Official title: Dissecting Mechanism of Neurofibromin Regulation in the Context of Sporadic Cancers and Neurofibromatosis Type 1
Funded at University of California, San Francisco · NIH-10949112 · Data updated 2026-11-12
This study is looking at a protein called neurofibromin to see how it affects people with Neurofibromatosis Type 1 (NF1) and certain cancers, and it aims to use a special gene-editing tool to fix problems in the NF1 gene, which could help improve treatment options and lower cancer risk for NF1 patients.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project start | December 4, 2024 |
| Project end | November 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $94,091 |
| NIH project number | 4K00CA274707-03 |
| Project ID | NIH-10949112 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Neurofibromatosis Type 1, particularly those with mutations in the NF1 gene.
✕Less likely to help
Patients without Neurofibromatosis Type 1 or those whose conditions are unrelated to NF1 mutations may not benefit from this research.
What this research studies
This research investigates the role of neurofibromin, a protein that regulates cell growth, in patients with Neurofibromatosis Type 1 (NF1) and sporadic cancers. The study aims to develop a CRISPR-based gene editing system to correct mutations in the NF1 gene, which could potentially restore normal function and reduce cancer risk. By focusing on the mechanisms of neurofibromin regulation, the research seeks to fill critical knowledge gaps that currently limit effective treatment options for NF1 patients. If successful, this approach could lead to innovative therapies that directly address the underlying genetic causes of NF1.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients with Neurofibromatosis Type 1 by correcting the genetic mutations that lead to cancer predisposition.
How similar studies have gone: While gene editing approaches have shown promise in other contexts, this specific application to NF1 is relatively novel and has not been extensively tested.
Where this research is happening
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University of California, San Francisco
San Francisco, United States Active
Researchers
- Principal investigator: Sitton, Madeleine Janette — University of California, San Francisco
- Study coordinator: Sitton, Madeleine Janette
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.