Creating a center to study the effects of gene knockouts in human cells
JAX MorPhiC Data Production Center
['FUNDING_OTHER'] · JACKSON LABORATORY · NIH-11098609
This study is exploring how turning off certain genes in human cells can help us understand their role in health and diseases, especially those affecting brain development, and it's designed for anyone interested in how our genes influence our bodies.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | JACKSON LABORATORY (nih funded) |
| Locations | 1 site (BAR HARBOR, UNITED STATES) |
| Trial ID | NIH-11098609 on ClinicalTrials.gov |
What this research studies
This research aims to establish a center focused on understanding how specific gene knockouts affect human cells, particularly in two important cell lineages: extra-embryonic and neuroectodermal. The team will use advanced techniques to engineer human induced pluripotent stem cells (iPSCs) and differentiate them into these lineages. By knocking out 250 protein-coding genes, the researchers will analyze the resulting cellular changes to gain insights into gene function and its implications for human health, especially in relation to neurodevelopmental disorders. This comprehensive approach combines expertise in molecular phenotyping and advanced cellular analysis.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic conditions or neurodevelopmental disorders that may be linked to specific gene functions.
Not a fit: Patients with conditions unrelated to the genes being studied or those who do not have a genetic component to their condition may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a better understanding of genetic contributions to various diseases, potentially informing new treatments and therapies.
How similar studies have performed: Other research has shown success in using gene knockout techniques to understand gene function, making this approach promising and not entirely novel.
Where this research is happening
BAR HARBOR, UNITED STATES
- JACKSON LABORATORY — BAR HARBOR, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: ROBSON, PAUL — JACKSON LABORATORY
- Study coordinator: ROBSON, PAUL
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.