Investigating genetic factors and drug discovery to improve brain function in Down syndrome
Large-scale Genetic Investigation and Drug Discovery to Counteract Down Syndrome Neural Deficits
This study is exploring how certain genes linked to Down syndrome affect brain development and behavior, using zebrafish to find new treatments that might help improve brain function for people with this condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project ID | NIH-11103227 on NIH RePORTER |
What this research studies
This research focuses on understanding the genetic influences of Down syndrome, which is the most common genetic cause of intellectual disability. By using zebrafish as a model organism, researchers will manipulate genes from chromosome 21 to observe their effects on brain development and behavior. The study aims to identify small molecules that could potentially prevent or reverse brain abnormalities associated with Down syndrome. This innovative approach combines genetic manipulation with high-throughput drug screening to find new therapeutic options.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Down syndrome, particularly those experiencing intellectual disabilities.
Not a fit: Patients without Down syndrome or those whose intellectual disabilities are not related to genetic factors on chromosome 21 may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that improve cognitive function and quality of life for individuals with Down syndrome.
How similar studies have performed: Previous research using zebrafish models has shown promise in identifying compounds that enter clinical trials, indicating potential success for this novel approach.
Where this research is happening
Worcester, United States
- Univ of Massachusetts Med Sch Worcester — Worcester, United States (Active)
Researchers
- Principal investigator: Thyme, Summer B — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Thyme, Summer B
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.