Making lab-grown brain cells behave like adult neurons
Engineering alternative splicing programs to accelerate maturation of stemcell-derived neurons
The team will reprogram how lab-grown neurons edit their RNA so they mature faster and better model age-related brain diseases for adults.
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-11241596 on NIH RePORTER |
What this research studies
This project uses human pluripotent stem cells to grow neurons in the lab and then modifies their alternative RNA splicing programs to push them from an immature, embryonic state toward adult-like neurons. Researchers will target key RNA-binding splicing factors and measure changes in gene and protein isoforms, cell shape, and electrical function to confirm maturation. The goal is to produce large numbers of mature, disease-relevant human neurons that reflect late-onset changes seen in neurodegenerative conditions. These improved cell models would be used to study disease mechanisms and speed development of therapies.
Who could benefit from this research
Good fit: Adults (21+) with age-related neurodegenerative diseases or healthy adult donors willing to provide cells or biological samples could potentially contribute samples to this work.
Not a fit: Patients seeking immediate clinical treatment or those with conditions unrelated to neurodegeneration are unlikely to gain direct medical benefit from this laboratory-focused project.
Why it matters
Potential benefit: If successful, this work could produce more accurate human neuron models that speed discovery of treatments for age-related neurodegenerative diseases.
How similar studies have performed: Previous research has used stem-cell-derived neurons and shown that alternative splicing matters for maturation, but deliberately engineering splicing programs to force adult-like maturation is a newer approach with encouraging early laboratory evidence.
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.