New lab tools to understand and treat ALS and related brain disorders
Innovating next generation technologies to define mechanisms of neurodegenerative disease and devise therapeutic strategies
['FUNDING_OTHER'] · STANFORD UNIVERSITY · NIH-11248759
Researchers will use advanced genetic tools to find new targets that could help people with ALS and related neurodegenerative diseases.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | STANFORD UNIVERSITY (nih funded) |
| Locations | 1 site (STANFORD, UNITED STATES) |
| Trial ID | NIH-11248759 on ClinicalTrials.gov |
What this research studies
If you have ALS or a related condition, this team is combining simple organisms and human cells to find genes that change levels of proteins that harm nerve cells. They use CRISPR to switch off or modify genes across the genome in human cells and follow up promising hits in mouse and cell models. The lab's prior discoveries—like targeting ataxin‑2—have already moved a therapy into early human trials, showing a path from lab work to patient testing. This project aims to discover more targets that could lead to new treatments over the coming years.
Who could benefit from this research
Good fit: People with ALS or frontotemporal disorders who want to follow research progress, consider donating samples, or be candidates for future clinical trials are the most relevant group.
Not a fit: Patients seeking immediate treatment are unlikely to benefit right away because this is primarily laboratory research and any therapies will take time to reach clinical use.
Why it matters
Potential benefit: Could identify new drug targets and approaches that eventually slow or stop progression of ALS and related disorders.
How similar studies have performed: Related approaches have shown promising preclinical results—reducing ataxin‑2 extended survival in mice and supported an ASO trial in humans—but many new targets remain untested in people.
Where this research is happening
STANFORD, UNITED STATES
- STANFORD UNIVERSITY — STANFORD, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: GITLER, AARON D. — STANFORD UNIVERSITY
- Study coordinator: GITLER, AARON D.
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.
Conditions: Amyotrophic Lateral Sclerosis Motor Neuron Disease