Protein balance problems and Alzheimer's-like dementia in Down syndrome
Proteostasis dysregulation and the development of Alzheimer's-like neurodegeneration and dementia in Down syndrome
This project asks whether disrupted protein management in the brain leads to Alzheimer's-like dementia in people with Down syndrome.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Temple Univ of the Commonwealth NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-11237975 on NIH RePORTER |
What this research studies
From the patient's perspective, researchers compare brain samples and markers from people with Down syndrome to those without to see how protein control systems are working. They study a well‑known Down syndrome mouse model (Ts65Dn) to follow early brain changes, measure mTOR pathway activity, and look at signs like amyloid plaques and synaptic loss. The team links biochemical findings to the pattern of neurodegeneration and tests whether correcting proteostasis-related pathways might prevent or slow those changes. Results could point to targets for future treatments aimed at the specific biology of Alzheimer's-like dementia in Down syndrome.
Who could benefit from this research
Good fit: Ideal candidates are people with Down syndrome (or their families) who can provide clinical information, consent to donate samples, or participate in related observational efforts connected to Temple University and collaborators.
Not a fit: People without Down syndrome or those with very advanced dementia unlikely to respond to early-targeted proteostasis interventions may not directly benefit from this work.
Why it matters
Potential benefit: If successful, this work could identify biological targets (like mTOR) that lead to therapies to delay or reduce Alzheimer's-like dementia in people with Down syndrome.
How similar studies have performed: Previous preclinical studies have linked mTOR and proteostasis to Alzheimer's pathology, but applying these findings specifically to Down syndrome is newer and less tested.
Where this research is happening
Philadelphia, United States
- Temple Univ of the Commonwealth — Philadelphia, United States (Active)
Researchers
- Principal investigator: Pratico, Domenico — Temple Univ of the Commonwealth
- Study coordinator: Pratico, Domenico
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.