Developing a system to manipulate RNA and proteins in specific neurons
Official title: Synthetic System for Neuron Subtype- and Context-Specific Subcellular RNA/Protein Manipulation and Bioactive Delivery
Funded at Harvard University · NIH-11038894 · Data updated 2026-11-12
This study is exploring a new way to control certain molecules in brain cells to better understand how they work and how problems with them might lead to conditions like autism and Huntington's disease, with the hope of finding new treatments.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Harvard University NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project start | September 20, 2024 |
| Project end | September 19, 2026 |
| Funding year | 2024 |
| Award (this year) | $481,250 |
| NIH project number | 1R21NS141110-01 |
| Project ID | NIH-11038894 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with autism spectrum disorder or Huntington's disease, as well as those with related neurological conditions.
✕Less likely to help
Patients with purely psychiatric disorders without a neurological component may not benefit from this research.
What this research studies
This research focuses on creating a synthetic biological system that can manipulate subcellular RNAs and proteins in specific types of neurons, particularly in growth cones and synapses. By using advanced techniques, the researchers aim to investigate how these molecules function within neuronal circuits and how their dysregulation may contribute to various neurological disorders. The approach includes integrating localization motifs with gene expression systems to enable precise control over RNA and protein levels in targeted neuronal areas. This could lead to new insights into neuronal connectivity and potential therapeutic strategies for conditions like autism and Huntington's disease.
Why it matters
Potential benefit: If successful, this research could lead to innovative therapies that specifically target and correct molecular dysfunctions in neurons, improving outcomes for patients with neurological disorders.
How similar studies have gone: Other research has shown promising results using similar synthetic biological systems to manipulate cellular functions, indicating a potential for success in this novel approach.
Where this research is happening
-
Harvard University
Cambridge, United States Active
Researchers
- Principal investigator: Macklis, Jeffrey D — Harvard University
- Study coordinator: Macklis, Jeffrey D
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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.