Developing a new method to edit genes in the brain using nanoparticles
Development of a Nanoparticle-Based Gene Editing Technology for Neurological Applications
This study is exploring a new way to deliver gene editing tools directly into the brain to help treat neurological diseases like Huntington's, Alzheimer's, and Parkinson's, with the hope of improving or even reversing the effects of these conditions for patients.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Ohio State University NIH-funded |
| Lab location | 1 site (Columbus, UNITED STATES) |
| Project ID | NIH-10669525 on NIH RePORTER |
What this research studies
This research focuses on creating a novel delivery system for CRISPR-based gene editing specifically targeting neurological diseases. By using a technique called convection-enhanced delivery (CED), the study aims to effectively and safely introduce gene editing components into brain cells through intracranial injections. The goal is to overcome existing challenges in delivering these components to large volumes of brain tissue, which is crucial for treating conditions like Huntington's, Alzheimer's, and Parkinson's disease. Patients may benefit from advancements in gene therapy that could potentially halt or reverse the progression of these debilitating diseases.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with neurological diseases such as Huntington's, Alzheimer's, or Parkinson's disease.
Not a fit: Patients with neurological conditions that are not caused by over-expression of pathogenic proteins may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to groundbreaking treatments for currently incurable neurological diseases.
How similar studies have performed: While CRISPR technology has shown promise in various applications, this specific approach to gene editing in the brain is innovative and has not been extensively tested in humans yet.
Where this research is happening
Columbus, UNITED STATES
- Ohio State University — Columbus, United States (Active)
Researchers
- Principal investigator: Bankiewicz, Krystof S — Ohio State University
- Study coordinator: Bankiewicz, Krystof S
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.