Understanding and treating inherited T cell defects
Inherited T cell defects: Diagnosis, Mechanisms and Treatments
This study is looking at how to better diagnose and treat inherited T cell problems, like severe combined immunodeficiency (SCID), by using cutting-edge technology to understand the genetics behind these conditions, so we can find the right treatments and catch issues early in newborns.
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-10929319 on NIH RePORTER |
What this research studies
This research focuses on diagnosing and treating inherited T cell defects, particularly severe combined immunodeficiency (SCID). It utilizes advanced techniques such as deep sequencing, genomic analyses, and CRISPR gene editing to explore how genetic sequences influence immune cell development. By integrating clinical data from patients with T cell insufficiencies, the research aims to identify causal mutations and develop targeted treatments. The program also leverages newborn screening methods to detect T cell deficiencies early in life, allowing for timely interventions.
Who could benefit from this research
Good fit: Ideal candidates for this research include infants diagnosed with severe combined immunodeficiency or other inherited T cell deficiencies.
Not a fit: Patients with acquired immune deficiencies or those without genetic causes for their immune issues may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to effective treatments for infants with inherited T cell defects, significantly improving their immune function and overall health.
How similar studies have performed: Previous research has shown promise in using gene editing techniques for treating genetic immunodeficiencies, indicating a potential for success in 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.