Molecular Gene Knockout Resource Center for Human iPSCs
Official title: JAX MorPhiC Data Production Center
Funded at Jackson Laboratory · NIH-11355419 · Data updated 2026-11-12
A national center will systematically create and study human iPSCs with 250 gene knockouts to uncover how these genes affect early development and neurodevelopment.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Jackson Laboratory NIH-funded |
| Lab location | 1 site (Bar Harbor, United States) |
| Project start | August 4, 2022 |
| Project end | May 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $1,874,508 |
| NIH project number | 5UM1HG012651-05 |
| Project ID | NIH-11355419 on NIH RePORTER |
Who could benefit from this research
✓Could help
Not directly a patient study; findings may inform future therapies for individuals with developmental gene disorders and neurodevelopmental conditions.
✕Less likely to help
This project does not involve direct patient participation or treatment during the study.
What this research studies
This project centers at The Jackson Laboratory for Genomic Medicine and builds a team to produce and characterize null alleles in human iPSCs. We will engineer knockouts of 250 protein-coding genes and differentiate the edited cells into extra-embryonic and neuroectodermal lineages, then perform deep phenotyping including single-cell analysis and metabolomics. The goal is to map how loss of each gene alters cellular function and development, with a focus on genes relevant to neurodevelopmental disorders. The findings could reveal new biological insights and potential targets for treating developmental diseases.
Why it matters
Potential benefit: If successful, it could reveal gene functions critical for human development and identify new avenues for understanding and treating neurodevelopmental disorders.
How similar studies have gone: Similar large-scale gene knockout projects have generated valuable functional maps in human cells, though this project’s combination of iPSC-derived lineages and deep phenotyping is relatively novel.
Where this research is happening
-
Jackson Laboratory
Bar Harbor, United States Active
Researchers
- Principal investigator: Robson, Paul — Jackson Laboratory
- Study coordinator: Robson, Paul
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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.