New methods for gene editing in the brain using engineered viruses
Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System
This study is exploring new ways to use specially designed viruses to safely deliver gene editing tools to the brain and spinal cord, which could lead to better treatments for genetic diseases that currently have no good options.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Broad Institute, INC. NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project ID | NIH-10490394 on NIH RePORTER |
What this research studies
This research focuses on developing advanced viral vectors, specifically engineered adeno-associated viruses (AAVs), to deliver gene editing tools throughout the central nervous system (CNS). By utilizing innovative techniques, the researchers aim to enhance the efficiency and safety of gene delivery, which is crucial for treating genetic diseases that currently lack effective therapies. The approach involves noninvasive methods to target neurons and astrocytes, potentially leading to significant advancements in gene therapy for CNS disorders. Patients may benefit from improved treatment options for conditions linked to genetic mutations affecting the CNS.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic disorders that impact the central nervous system and for whom current treatments are ineffective.
Not a fit: Patients with non-genetic neurological conditions or those whose conditions do not involve the central nervous system may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide new, effective treatment options for patients with genetic diseases affecting the central nervous system.
How similar studies have performed: Previous research has shown promise in using engineered AAVs for gene delivery, indicating potential for success in this novel approach.
Where this research is happening
Cambridge, United States
- Broad Institute, INC. — Cambridge, United States (Active)
Researchers
- Principal investigator: Deverman, Benjamin E — Broad Institute, INC.
- Study coordinator: Deverman, Benjamin E
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.