Testing new genome editing techniques for treating severe diseases.
BCM/RICE GENOME EDITING TESTING CENTER
This study is exploring new ways to fix genes in cells that could help treat serious diseases, using specially modified mice to make sure the methods are safe and work well before trying them in people.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Baylor College of Medicine NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project ID | NIH-10917288 on NIH RePORTER |
What this research studies
This research focuses on advancing somatic cell genome editing, which has the potential to cure severe human diseases. It utilizes genetically modified mice to assess the effectiveness and safety of these editing techniques before they are tested in humans. By employing fluorescent reporter mice, researchers can visualize and quantify the editing events at a cellular level, ensuring that the methods are both effective and safe. The project aims to consolidate various expertise in molecular biology, bioengineering, and immunology to create a comprehensive testing center for these innovative therapies.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with severe genetic diseases that may benefit from genome editing therapies.
Not a fit: Patients with conditions that are not genetic in nature or those who are not eligible for genome editing therapies may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to groundbreaking treatments for severe diseases that currently have limited options.
How similar studies have performed: Previous research has shown promise in using genome editing techniques in animal models, indicating potential for success in this approach.
Where this research is happening
Houston, United States
- Baylor College of Medicine — Houston, United States (Active)
Researchers
- Principal investigator: Heaney, Jason D. — Baylor College of Medicine
- Study coordinator: Heaney, Jason D.
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.