Investigating genetic factors and drug discovery to improve brain function in Down syndrome
Official title: Large-scale Genetic Investigation and Drug Discovery to Counteract Down Syndrome Neural Deficits
Funded at Univ of Massachusetts Med Sch Worcester · NIH-11103227 · Data updated 2026-11-12
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 start | June 5, 2024 |
| Project end | May 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $837,498 |
| NIH project number | 5R01HD115159-02 |
| Project ID | NIH-11103227 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Down syndrome, particularly those experiencing intellectual disabilities.
✕Less likely to help
Patients without Down syndrome or those whose intellectual disabilities are not related to genetic factors on chromosome 21 may not benefit from this research.
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.
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 gone: 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
-
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
Related research
Other NIH-funded projects on the same topics.
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Exploring how Down syndrome affects optic nerve health in kids
A study using a mouse model to understand why optic nerve problems are common in children with Down syndrome and how to prevent them.
-
Executive Function Enhancement for Young Children with Down Syndrome
A caregiver-guided program for kids with Down syndrome aged up to 11 to strengthen their thinking skills during early childhood.
-
Silencing an Extra Chromosome to Help Down Syndrome and Related Conditions
A patient-friendly plan to use a gene-based approach to quiet an extra chromosome in cells and a mouse model to reduce disease-related effects in Down syndrome and other chromosomal imbalance disorders.
-
Infant Down Syndrome Brain and Behavior Study
A study of infants with Down syndrome to collect brain imaging and developmental data, creating a large, shared resource to understand how autism and other factors relate to development.
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DS-ARC: Smartphone-based cognitive check for people with Down syndrome at risk for Alzheimer’s
A smartphone-based cognitive test is being developed and tested for people with Down syndrome who may develop Alzheimer’s disease, to track changes quietly in daily life.
-
Immune and NGF factors in Alzheimer’s for adults with Down syndrome
This grant studies why people with Down syndrome develop Alzheimer’s disease by looking at immune signals and nerve growth factor problems.
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.