Targeting brain support cells to treat neurological diseases
Official title: Glia Exclusive Gene Therapy
Funded at University of Pittsburgh at Pittsburgh · NIH-10881987 · Data updated 2026-11-12
This study is exploring a new way to use gene therapy to help brain support cells, called glial cells, which are important in conditions like Alzheimer's and autism, by delivering special RNA molecules that won't upset your immune system.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pittsburgh at Pittsburgh NIH-funded |
| Lab location | 1 site (Pittsburgh, United States) |
| Project start | July 15, 2023 |
| Project end | June 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $198,750 |
| NIH project number | 5R21EB033121-02 |
| Project ID | NIH-10881987 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with Alzheimer's disease, autism spectrum disorders, or other neurological conditions where glial cells are implicated.
✕Less likely to help
Patients with neurological conditions not related to glial cell dysfunction may not benefit from this research.
What this research studies
This research investigates a new approach to gene therapy that focuses on glial cells, which are crucial support cells in the brain involved in various neurological diseases like Alzheimer's and autism. The team aims to develop a novel method to deliver genes specifically to these glial cells using engineered RNA molecules called ModRNAs, which are designed to avoid triggering immune responses. By customizing these ModRNAs to target specific types of glial cells, the research seeks to enhance the effectiveness of gene therapy for conditions where glial dysfunction plays a significant role.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for neurological diseases by specifically targeting the underlying cellular mechanisms in glial cells.
How similar studies have gone: While gene therapy has shown promise in targeting neurons, this specific approach to exclusively target glial cells is novel and has not been extensively tested in prior research.
Where this research is happening
-
University of Pittsburgh at Pittsburgh
Pittsburgh, United States Active
Researchers
- Principal investigator: Shemesh, Or — University of Pittsburgh at Pittsburgh
- Study coordinator: Shemesh, Or
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.