Mouse models to find treatments for thinking and memory problems in Down syndrome

Generation and analysis of new mouse models to determine novel therapeutic targets for Down syndrome-associated cognitive deficits

['FUNDING_R01'] · ROSWELL PARK CANCER INSTITUTE CORP · NIH-11329125

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

Phase['FUNDING_R01']
Study typeNih_funding
SexAll
SponsorROSWELL PARK CANCER INSTITUTE CORP (nih funded)
Locations1 site (BUFFALO, UNITED STATES)
Trial IDNIH-11329125 on ClinicalTrials.gov

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.

Who could benefit from this research

Good fit: 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.

Not a fit: 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.

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 performed: 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

BUFFALO, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.

View on NIH RePORTER →

Conditions: Alzheimer disease dementia, Alzheimer syndrome, Alzheimer's Disease

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.