Precision gene editing for motor neuron diseases
Precision Base Editing for the Treatment of Motor Neuron Diseases
This project aims to create precise gene-editing tools to fix single-letter DNA errors that cause motor neuron diseases like ALS and SMA in adults.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-11128756 on NIH RePORTER |
What this research studies
Researchers are building and testing ‘base editors,’ gene-editing tools that can directly correct single-letter DNA changes linked to motor neuron diseases. They will screen many combinations of editing enzymes, Cas proteins, and guide RNAs to find the safest and most accurate editors for different mutations. The team will use lab models and AAV delivery methods to see which editors best reach and correct motor neurons. Successful lab findings would guide development of tailored therapies for people with ALS, SMA, and related conditions.
Who could benefit from this research
Good fit: Adults with genetically confirmed motor neuron diseases (such as ALS or SMA) caused by single-letter DNA changes would be the most likely candidates for future therapies.
Not a fit: People whose conditions are not caused by the specific DNA changes targeted, or those with extensive irreversible nerve damage, may not benefit from these approaches.
Why it matters
Potential benefit: If successful, this work could lead to precise gene therapies that correct disease-causing DNA errors and slow or stop motor neuron degeneration.
How similar studies have performed: Laboratory studies have shown base editors can correct single-letter DNA errors in cells and animals, but translating these methods to safe, predictable treatments for human motor neurons is still novel and experimental.
Where this research is happening
Boston, United States
- Boston Children's Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Arbab, Mandana — Boston Children's Hospital
- Study coordinator: Arbab, Mandana
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.