Understanding how gene loss affects human health
Center for scalable knockout and multimodal phenotyping in genetically diverse human genomes
This study is looking at how losing different genes affects our bodies and health, using special techniques to create a library of cells from various backgrounds, which could help us understand conditions like autism and diabetes better and find new treatments for patients.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Sloan-Kettering Inst Can Research NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11099976 on NIH RePORTER |
What this research studies
This research aims to explore the function of every human gene by creating a detailed catalog of how gene loss impacts various biological traits. It utilizes advanced techniques like gene editing and multicellular systems to study the effects of gene knockouts in diverse human genetic backgrounds. By focusing on human pluripotent stem cells, the research will generate a repository of cell lines that represent different ancestral populations, which will be used to investigate conditions such as autism and diabetes. Patients may benefit from insights gained about their genetic conditions and potential treatments.
Who could benefit from this research
Good fit: Ideal candidates for participation or benefit include individuals with neurodevelopmental or metabolic disorders, particularly those with a family history of autism or diabetes.
Not a fit: Patients with conditions unrelated to genetic factors or those not represented in the diverse genetic backgrounds being studied may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new understanding and treatments for genetic disorders, including autism and diabetes.
How similar studies have performed: Previous research using gene editing and stem cell technologies has shown promise in understanding genetic disorders, indicating that this approach could yield valuable insights.
Where this research is happening
New York, United States
- Sloan-Kettering Inst Can Research — New York, United States (Active)
Researchers
- Principal investigator: Huangfu, Danwei — Sloan-Kettering Inst Can Research
- Study coordinator: Huangfu, Danwei
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.