Finding DNA switches that control T cells and autoimmune risk
Multiscale genome engineering to map cis-regulatory variants in human and mouse
['FUNDING_R01'] · NEW YORK GENOME CENTER · NIH-11135344
Using gene-editing and single-cell methods to find DNA 'switches' that change how T cells behave for people with autoimmune conditions like brittle diabetes.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | NEW YORK GENOME CENTER (nih funded) |
| Locations | 1 site (NEW YORK, UNITED STATES) |
| Trial ID | NIH-11135344 on ClinicalTrials.gov |
What this research studies
Researchers will use CRISPR gene-editing tools on primary human T cells and in mouse models to test thousands of noncoding DNA regions tied to autoimmune risk. They will run loss- and gain-of-function screens across nine T-cell behaviors, then apply single-cell profiling and saturation mutagenesis to pinpoint regulatory elements that change gene activity. The team will focus on candidate regions overlapping GWAS signals and build context-specific enhancer maps for T cells by combining human genetics, immunology, and computational modeling. The work links DNA differences to immune cell function using donor blood samples and laboratory models.
Who could benefit from this research
Good fit: Ideal participants would be people with autoimmune conditions (for example type 1/brittle diabetes) or healthy donors willing to give blood samples for T-cell research.
Not a fit: Patients seeking immediate changes to their treatment are unlikely to gain direct clinical benefit because this is foundational research focused on informing future therapies rather than providing immediate interventions.
Why it matters
Potential benefit: If successful, this could reveal specific DNA elements that drive autoimmune reactions and help guide new diagnostics or targeted therapies for conditions like brittle diabetes.
How similar studies have performed: Related CRISPR-based screens and enhancer-mapping studies have identified functional regulatory elements in immune cells, but applying these methods at large scale to many GWAS loci for autoimmune disease is relatively new.
Where this research is happening
NEW YORK, UNITED STATES
- NEW YORK GENOME CENTER — NEW YORK, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: SANJANA, NEVILLE — NEW YORK GENOME CENTER
- Study coordinator: SANJANA, NEVILLE
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.
Conditions: Autoimmune Diseases, Brittle Diabetes Mellitus