Understanding how inherited prion diseases affect the brain
Mechanisms of Neurodegeneration in Hereditary Prion Diseases
This study is looking at how certain gene changes related to hereditary prion diseases affect brain cells, using special lab-grown cells to better understand how these changes might lead to problems like Alzheimer's disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston University Medical Campus NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-11168162 on NIH RePORTER |
What this research studies
This research investigates the cellular and molecular mechanisms behind hereditary prion diseases, particularly focusing on how mutations in the PRNP gene lead to neurodegeneration. By utilizing innovative induced pluripotent stem cell (iPSC) technology, the study aims to analyze the effects of mutant prion proteins on human neurons and glial cells. The goal is to uncover the toxic properties of these proteins and their interactions within the brain, which could provide insights into the progression of related conditions like Alzheimer's disease.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals with a family history of hereditary prion diseases or specific mutations in the PRNP gene.
Not a fit: Patients with sporadic forms of prion diseases or those without genetic mutations related to prion disorders may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic strategies for treating neurodegenerative diseases linked to prion mutations.
How similar studies have performed: Previous research has shown promise in understanding prion diseases through animal models, but this approach using human-derived cells is relatively novel.
Where this research is happening
Boston, United States
- Boston University Medical Campus — Boston, United States (Active)
Researchers
- Principal investigator: Harris, David a — Boston University Medical Campus
- Study coordinator: Harris, David a
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.