Mapping what essential human genes do
Molecular and cellular characterization of essential human genes.
['FUNDING_OTHER'] · NORTHWESTERN UNIVERSITY · NIH-11143693
This project builds a large catalog showing what happens to human cells when key genes are switched off, to help people with genetic heart and other diseases.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | NORTHWESTERN UNIVERSITY (nih funded) |
| Locations | 1 site (CHICAGO, UNITED STATES) |
| Trial ID | NIH-11143693 on ClinicalTrials.gov |
What this research studies
Researchers will edit human induced pluripotent stem cells with CRISPR to add a tiny switch (an auxin-inducible degron) to many genes. When the switch is triggered with a small chemical called auxin, the targeted protein is rapidly removed, creating a reversible 'off' state for that gene in cells. Each edited gene also gets a barcode so scientists can track the effects of hundreds of thousands of gene 'turn-offs' across different cell types and organoid models that mimic the heart and other tissues. The goal is to map the molecular and cellular consequences of missing genes to help guide future diagnosis and treatments for genetic diseases.
Who could benefit from this research
Good fit: People with known or suspected genetic heart conditions, or those interested in contributing samples for genetic research, would be the most relevant candidates for future related studies.
Not a fit: Patients without genetic causes for their condition or those seeking immediate treatment are unlikely to get direct benefit from this lab-focused project.
Why it matters
Potential benefit: If successful, this work could reveal which genes cause or contribute to heart and other diseases and point scientists toward new tests or therapies.
How similar studies have performed: Components like CRISPR editing, auxin-inducible degrons, and hiPSC-derived organoids have worked in labs, but combining them at large scale to catalog every gene is new.
Where this research is happening
CHICAGO, UNITED STATES
- NORTHWESTERN UNIVERSITY — CHICAGO, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: ADLI, MAZHAR — NORTHWESTERN UNIVERSITY
- Study coordinator: ADLI, MAZHAR
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: Cardiovascular Diseases