How an extra APP gene affects brain growth and Alzheimer’s in Down syndrome
The role of APP in neurogenesis and AD in Down syndrome
This work looks at whether having an extra copy of the APP gene harms brain cell growth and leads to Alzheimer’s changes in people with Down syndrome.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Illinois at Chicago NIH-funded |
| Lab location | 1 site (Chicago, UNITED STATES) |
| Project ID | NIH-11297675 on NIH RePORTER |
What this research studies
Researchers are using stem cells from people with Down syndrome to grow tiny brain-like organoids that model early brain development. They use CRISPR gene editing to remove one copy of the APP gene in these cells and compare organoids with and without the extra APP copy. The team will examine neural stem cell proliferation, neuron formation, cortical layering, and early signs of Alzheimer’s pathology such as amyloid accumulation. The aim is to find out if APP overdose drives the neurodevelopmental problems and Alzheimer’s changes seen in Down syndrome.
Who could benefit from this research
Good fit: People with Down syndrome (or their legal guardians) who can provide consent to donate blood or skin samples for iPSC generation would be ideal candidates to contribute samples for this research.
Not a fit: People without Down syndrome or those seeking immediate clinical treatments should not expect direct benefits from this lab-based, preclinical work.
Why it matters
Potential benefit: If successful, this work could point to APP as a target for treatments that prevent or delay Alzheimer’s changes and improve brain development in people with Down syndrome.
How similar studies have performed: Prior cell and organoid research has linked APP to neuronal development and Aβ production, but using CRISPR to remove an APP copy in Down syndrome-derived organoids is relatively new.
Where this research is happening
Chicago, UNITED STATES
- University of Illinois at Chicago — Chicago, United States (Active)
Researchers
- Principal investigator: Lazarov, Orly — University of Illinois at Chicago
- Study coordinator: Lazarov, Orly
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 below.