Gene therapy for disorders caused by mutations in collagen genes
Official title: Gene therapy for disorders of the extracellular matrix
Funded at University of California, San Francisco · NIH-10658481 · Data updated 2026-11-12
This study is exploring new gene therapy methods to help people with cerebrovascular diseases caused by changes in specific genes, aiming to fix these genetic issues at their source and improve treatments for conditions like Gould syndrome.
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 | April 1, 2023 |
| Project end | March 31, 2026 |
| Funding year | 2023 |
| Award (this year) | $2,481,348 |
| NIH project number | 1RF1NS128217-01A1 |
| Project ID | NIH-10658481 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with Gould syndrome or those with COL4A1 and COL4A2 mutations experiencing cerebrovascular complications.
✕Less likely to help
Patients without COL4A1 or COL4A2 mutations or those with unrelated cerebrovascular diseases may not benefit from this research.
What this research studies
This research focuses on developing gene therapy techniques to address disorders caused by mutations in the COL4A1 and COL4A2 genes, which lead to a range of cerebrovascular diseases. By utilizing advanced genome editing technologies like CRISPR and novel viral vectors, the project aims to correct the genetic defects at their source. The research will involve testing these therapeutic approaches on patient-derived cells and animal models to gather crucial pre-clinical data. This innovative approach seeks to provide a targeted intervention for patients suffering from Gould syndrome and related conditions.
Why it matters
Potential benefit: If successful, this research could lead to groundbreaking treatments that correct the underlying genetic causes of cerebrovascular diseases associated with Gould syndrome.
How similar studies have gone: Previous research has shown promise in using genome editing technologies for similar genetic disorders, indicating potential for success in this novel approach.
Where this research is happening
-
University of California, San Francisco
San Francisco, United States Active
Researchers
- Principal investigator: Gould, Douglas — University of California, San Francisco
- Study coordinator: Gould, Douglas
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.