Personalized antisense therapy for ultra-rare ALS
Silence ALS: A Platform for the Discovery and Development of Antisense Therapeutics for Patients with Ultra-Rare Forms of ALS
Personalized antisense medicines designed to block harmful, ultra-rare gene mutations that cause ALS in people with those mutations.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11312678 on NIH RePORTER |
What this research studies
This project aims to create individualized antisense oligonucleotide (ASO) treatments that specifically target dominant, disease-causing mutations in the TARDBP gene linked to ALS. Scientists at Columbia University and a nonprofit partner will design allele-specific ASOs, test them in laboratory models and patient-derived materials, and develop the safety and manufacturing steps needed for clinical use. The effort focuses on ultra-rare mutations and uses a precision-medicine approach to expand access to gene-based therapies for patients who currently have few options. If preclinical results are promising, the team plans to prepare these therapies so they could eventually be offered to eligible patients.
Who could benefit from this research
Good fit: People with ALS who have a confirmed, dominant disease-causing mutation in TARDBP or a similar ultra-rare, targetable allele and who can provide genetic confirmation and possibly biological samples.
Not a fit: People with sporadic ALS without a confirmed, targetable genetic mutation, or with mutations not addressable by allele-specific ASOs, are unlikely to benefit from this program.
Why it matters
Potential benefit: If successful, this work could produce individualized treatments that slow or stop disease progression for patients with specific ultra-rare ALS gene mutations.
How similar studies have performed: Antisense therapies have shown success in other neurological diseases and in early ALS programs (for example SOD1 and C9orf72), so the approach is promising though allele-specific TARDBP treatments are novel.
Where this research is happening
New York, United States
- Columbia University Health Sciences — New York, United States (Active)
Researchers
- Principal investigator: Shneider, Neil Alan — Columbia University Health Sciences
- Study coordinator: Shneider, Neil Alan
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.