Using mouse models to find gene targets for improving thinking and learning in Down syndrome

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

NIH-funded research Roswell Park Cancer Institute Corp · NIH-11178435

Researchers are using new mouse models to find specific genes that could be targeted to help people with Down syndrome think and learn better.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionRoswell Park Cancer Institute Corp NIH-funded
Lab location1 site (Buffalo, United States)
Project IDNIH-11178435 on NIH RePORTER

What this research studies

This project creates and studies new mouse models that mimic parts of human chromosome 21 linked to Down syndrome. Scientists will change gene copy number and use genetic tools to see which genes affect brain development, structure, and behavior. They will combine behavioral tests with brain tissue analyses to pinpoint dosage-sensitive genes tied to learning and memory problems. The goal is to identify specific gene targets that could guide future therapies for cognitive difficulties in Down syndrome.

Who could benefit from this research

Good fit: People with Down syndrome and their caregivers who are interested in future treatments based on gene-targeted approaches would be most relevant to follow this work.

Not a fit: Patients seeking immediate treatments or current clinical trial enrollment are unlikely to benefit directly because the work is preclinical in mice.

Why it matters

Potential benefit: If successful, the work could point to specific genes to target with therapies that improve learning and memory for people with Down syndrome.

How similar studies have performed: Earlier mouse-based studies have identified candidates such as DYRK1A and provided promising leads, but turning those findings into effective human therapies has been limited so far.

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.
Conditions Bartholin-Patau syndromeCandidate Disease Gene
Last reviewed 2026-06-15 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.