Targeting microglia to treat brain immune and infectious disorders
Unlocking microglia targeting for neurotherapeutics
This project tests ways to change brain immune cells (microglia) so they can be used to treat people with immune-related or infectious brain conditions.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-11169957 on NIH RePORTER |
What this research studies
Researchers are working to make microglia — the brain's resident immune cells — amenable to gene modification so they can be used as targeted therapies. In the lab they will study antiviral pathways that stop lentiviruses from entering microglia using cultured cells and genetically modified mice. The team will run a focused CRISPR knockout screen with new AAV tools that preferentially target microglia to find genes that block viral delivery. Findings aim to enable safe gene editing of microglia and improve understanding of microglia-driven antiviral immunity.
Who could benefit from this research
Good fit: People with neuroimmune disorders, genetic leukodystrophies, interferon-related brain diseases, or HIV-related neurological complications are the types of patients who might benefit from this line of research in the future.
Not a fit: Patients seeking immediate therapies or those with conditions unrelated to the brain or immune system would not see direct benefit from this early laboratory research.
Why it matters
Potential benefit: If successful, this work could open the door to microglia-based gene therapies for neuroimmune, genetic, and infectious brain disorders.
How similar studies have performed: Gene therapy approaches have succeeded in other brain cell types, but genetically modifying microglia with lentiviruses is largely novel and this proposal includes the first CRISPR screen in primary microglia.
Where this research is happening
Philadelphia, United States
- Children's Hosp of Philadelphia — Philadelphia, United States (Active)
Researchers
- Principal investigator: Bennett, Mariko L. — Children's Hosp of Philadelphia
- Study coordinator: Bennett, Mariko L.
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.