Turning back on early motor neuron genes to protect against ALS
Effects of Re-expression of Motor Neuron Selector Transcription Factors on ALS Pathology
Researchers will re-activate two early-life motor neuron genes to try to make motor neurons more resistant to damage in ALS.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11238416 on NIH RePORTER |
What this research studies
If you have ALS, this project will put two embryonic motor neuron genes (ISL1 and LHX3) back into mature motor neurons in mouse ALS models and into motor neurons made from patient-derived iPSC cells in the lab. The team will watch whether this re-expression improves neuron survival, reduces protein-folding stress, and lessens disease signs in mice. They will also measure how gene activity, chromatin accessibility, and DNA methylation change when these genes are turned back on. Together, these steps aim to show whether returning motor neurons to a more youthful state can protect them and point toward future therapies.
Who could benefit from this research
Good fit: Adults with ALS who are willing to donate tissue or cells for iPSC generation or who may join future clinical trials based on this work would be the most relevant participants.
Not a fit: People looking for immediate treatment changes are unlikely to benefit now because the work is preclinical and done in mice and lab-grown cells.
Why it matters
Potential benefit: If successful, this approach could identify new ways to strengthen motor neurons and slow ALS-related neuron loss.
How similar studies have performed: This is a relatively novel approach for ALS with limited prior success, though related reprogramming and developmental-factor strategies have shown protective effects in some other neurodegeneration models.
Where this research is happening
New York, United States
- Columbia University Health Sciences — New York, United States (Active)
Researchers
- Principal investigator: Wichterle, Hynek — Columbia University Health Sciences
- Study coordinator: Wichterle, Hynek
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.