Developing a treatment for ALS and frontotemporal dementia using UNC13A antisense oligonucleotides
Development of an UNC13A antisense oligonucleotide treatment for ALS and FTD
This study is testing a new treatment for people with ALS and frontotemporal dementia that aims to help by targeting a specific gene, which could improve brain health and slow down the progression of these conditions.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Acurastem, INC. NIH-funded |
| Lab location | 1 site (Pasadena, UNITED STATES) |
| Project ID | NIH-10914945 on NIH RePORTER |
What this research studies
This research focuses on creating a new treatment for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) by using antisense oligonucleotides to target the UNC13A gene. The approach aims to suppress the inclusion of cryptic exons in UNC13A mRNA, which is linked to the progression of these diseases. By addressing the underlying genetic factors associated with ALS and FTD, the treatment could potentially restore normal protein function and improve neuronal health. Patients will be monitored for changes in symptoms and overall health as the treatment is developed and tested.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults diagnosed with ALS or FTD, particularly those with genetic risk factors related to the UNC13A gene.
Not a fit: Patients with other forms of neurodegenerative diseases that do not involve TDP-43 pathology may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a novel therapy that slows the progression of ALS and FTD, improving quality of life for patients.
How similar studies have performed: Previous research has shown promise in targeting genetic factors in neurodegenerative diseases, suggesting that this approach could be effective.
Where this research is happening
Pasadena, UNITED STATES
- Acurastem, INC. — Pasadena, United States (Active)
Researchers
- Principal investigator: Chang, Wen-Hsuan — Acurastem, INC.
- Study coordinator: Chang, Wen-Hsuan
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.