Targeting neurons to change gene expression using specialized antibodies
Official title: Neuron-specific modulation of gene expression using systemically administered bispecific antibody-ASO conjugates
Funded at University of Michigan at Ann Arbor · NIH-11127778 · Data updated 2026-11-12
This study is exploring a new way to deliver special treatments directly to brain cells using a friendly antibody, making it easier and safer for people with neurological conditions to receive help without needing invasive procedures.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Michigan at Ann Arbor NIH-funded |
| Lab location | 1 site (Ann Arbor, United States) |
| Project start | July 1, 2024 |
| Project end | May 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $609,153 |
| NIH project number | 5R01NS138455-02 |
| Project ID | NIH-11127778 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research would be individuals with neurological conditions that could benefit from targeted gene modulation.
✕Less likely to help
Patients with conditions unrelated to neuronal gene expression or those who do not have access to the required treatment facilities may not benefit from this research.
What this research studies
This research focuses on developing a new method to deliver anti-sense oligonucleotides (ASOs) directly to neurons in the brain through a specialized bispecific antibody. By using this approach, the researchers aim to overcome the limitations of traditional methods that require invasive procedures and have poor specificity. The goal is to enable the modulation of gene expression in neurons via a non-invasive intravenous administration, which could lead to better understanding and treatment of various neurological conditions.
Why it matters
Potential benefit: If successful, this research could provide a novel and less invasive way to treat neurological disorders by precisely targeting gene expression in neurons.
How similar studies have gone: While the use of ASOs has been explored, this specific approach of using bispecific antibodies for targeted delivery to neurons is novel and has not been extensively tested.
Where this research is happening
-
University of Michigan at Ann Arbor
Ann Arbor, United States Active
Researchers
- Principal investigator: Thurber, Greg — University of Michigan at Ann Arbor
- Study coordinator: Thurber, Greg
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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.