Investigating brain cell dysfunction in Down Syndrome using advanced cell models
Official title: Understanding neuronal dysfunction in Down Syndrome using assembloids and xenotransplanted cortical organoids
Funded at Boston University Medical Campus · NIH-10944356 · Data updated 2026-11-12
This study is looking at how Down Syndrome affects brain cells and can lead to memory problems and a higher chance of developing Alzheimer's, using special lab-grown brain cells to better understand these challenges in a friendly and relatable way.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston University Medical Campus NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 30, 2024 |
| Project end | August 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $2,357,335 |
| NIH project number | 1RF1AG088529-01 |
| Project ID | NIH-10944356 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with Down Syndrome, particularly those experiencing cognitive challenges.
✕Less likely to help
Patients without Down Syndrome or those who do not exhibit cognitive impairments may not benefit from this research.
What this research studies
This research focuses on understanding how Down Syndrome, caused by an extra copy of chromosome 21, leads to cognitive challenges and increases the risk of Alzheimer's disease. The team will create specialized brain cell models from induced pluripotent stem cells to study how these cells behave and connect in individuals with Down Syndrome. By using advanced techniques like calcium imaging, they aim to uncover the underlying mechanisms of neuronal dysfunction that contribute to cognitive deficits. This approach allows for a more accurate representation of human brain activity compared to traditional animal models.
Why it matters
Potential benefit: If successful, this research could lead to new insights and potential therapies for cognitive impairments associated with Down Syndrome and Alzheimer's disease.
How similar studies have gone: While this approach is innovative, similar studies using advanced cellular models have shown promise in understanding other neurological conditions.
Where this research is happening
-
Boston University Medical Campus
Boston, United States Active
Researchers
- Principal investigator: Zeldich, Ella — Boston University Medical Campus
- Study coordinator: Zeldich, Ella
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.