Zebrafish models for rare genetic diseases
Resource of zebrafish models of human diseases
['FUNDING_OTHER'] · UNIVERSITY OF OREGON · NIH-11184266
The team will create zebrafish versions of patients' gene changes to help understand rare genetic conditions for people with undiagnosed or suspected inherited disorders.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF OREGON (nih funded) |
| Locations | 1 site (EUGENE, UNITED STATES) |
| Trial ID | NIH-11184266 on ClinicalTrials.gov |
What this research studies
Researchers will work with diagnostic centers to use patients' genetic and clinical information to select likely disease-causing variants. They will run bioinformatics pipelines to prioritize variants and then use CRISPR-based gene editing to create 'humanized' zebrafish that carry those same changes. Each model will be validated by DNA sequencing and by checking for physical and molecular traits that mirror the patient's condition. The validated zebrafish lines will be deposited in the Zebrafish International Resource Center so other scientists can use them to speed diagnosis and treatment research.
Who could benefit from this research
Good fit: Ideal candidates are people with undiagnosed or suspected rare genetic disorders whose DNA variants are identified by participating diagnostic centers.
Not a fit: People with common non-genetic conditions or those whose genetic changes are not amenable to zebrafish modeling are unlikely to benefit directly from this project.
Why it matters
Potential benefit: If successful, this resource could help link specific genetic variants to disease, speeding diagnosis and supporting development of treatments for people with rare conditions.
How similar studies have performed: Zebrafish and other model organisms have often helped clarify the effects of human gene variants, though assembling a large, standardized public library of humanized models is a newer effort.
Where this research is happening
EUGENE, UNITED STATES
- UNIVERSITY OF OREGON — EUGENE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: WESTERFIELD, MONTE — UNIVERSITY OF OREGON
- Study coordinator: WESTERFIELD, MONTE
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: Animal Disease Models