Managing a program to identify and treat genetic disorders in patients with immune deficiencies
Administrative Core
This study is all about finding and fixing the genetic issues that cause severe combined immunodeficiency (SCID) and T cell deficiencies, so we can create personalized treatments to help those affected feel better and stay healthy.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-10929320 on NIH RePORTER |
What this research studies
This research focuses on managing a comprehensive program that combines bioinformatics and genomic expertise to identify genetic variants causing severe combined immunodeficiency (SCID) and T cell deficiencies. The program aims to utilize genome editing techniques to develop personalized treatment approaches for affected individuals. It includes systematic identification of disease-causing variants, genome editing to correct these variants, and functional analysis to understand their impact. The administration core will oversee the scientific and financial management of the program, ensuring effective collaboration among researchers.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with severe combined immunodeficiency (SCID) or T cell deficiencies.
Not a fit: Patients with immune deficiencies not related to genetic variants or those who do not have SCID or T cell deficiencies may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to personalized treatments for patients with genetic immune deficiencies, improving their health outcomes.
How similar studies have performed: Previous research has shown promise in using genome editing for genetic disorders, indicating potential success for this approach.
Where this research is happening
San Francisco, United States
- University of California, San Francisco — San Francisco, United States (Active)
Researchers
- Principal investigator: Marson, Alexander — University of California, San Francisco
- Study coordinator: Marson, Alexander
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.