Mouse models to find treatments for thinking and memory problems in Down syndrome
Official title: Generation and analysis of new mouse models to determine novel therapeutic targets for Down syndrome-associated cognitive deficits
Funded at Roswell Park Cancer Institute Corp · NIH-11329125 · Data updated 2026-11-12
Researchers will build new mouse models that mimic Down syndrome to discover genes and pathways that could lead to treatments for thinking and memory problems in people with Down syndrome.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Roswell Park Cancer Institute Corp NIH-funded |
| Lab location | 1 site (Buffalo, United States) |
| Project start | September 13, 2022 |
| Project end | August 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $116,033 |
| NIH project number | 3R01HD109750-04S1 |
| Project ID | NIH-11329125 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with Down syndrome, their families, or caregivers who want to contribute clinical data, biospecimens, or join future human studies based on these findings would be ideal candidates for follow‑up participation.
✕Less likely to help
People looking for immediate treatments or clinical care are unlikely to benefit directly because this is preclinical mouse research that may take years to translate to human therapies.
What this research studies
This project makes new mouse versions of the extra chromosome regions found in Down syndrome using advanced chromosome‑engineering tools. Scientists will combine duplications and targeted deletions to pinpoint which extra genes cause learning and memory problems. They will study brain biology and behavior in these mice to link specific genes or pathways to cognitive issues. The goal is to highlight targets that drug developers or future clinical studies could pursue to help people with Down syndrome.
Why it matters
Potential benefit: If successful, the work could identify specific genes or biological pathways to target with therapies that improve learning and memory in people with Down syndrome.
How similar studies have gone: Mouse models with extra copies of chromosome 21 regions have previously helped identify candidate genes and pathways linked to cognition, but translating those findings into effective human treatments remains challenging.
Where this research is happening
-
Roswell Park Cancer Institute Corp
Buffalo, United States Active
Researchers
- Principal investigator: Yu, Eugene — Roswell Park Cancer Institute Corp
- Study coordinator: Yu, Eugene
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.
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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.
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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.
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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.