Developing RNA aptamers to inhibit AMPA receptors for ALS treatment
Animal testing of RNA aptamers as ALS drug candidate
['FUNDING_R01'] · STATE UNIVERSITY OF NEW YORK AT ALBANY · NIH-11083091
This study is looking at a new type of treatment for ALS by testing special RNA molecules that might help slow down the disease in mice, with hopes of finding a safer and more effective option for people living with ALS.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | STATE UNIVERSITY OF NEW YORK AT ALBANY (nih funded) |
| Locations | 1 site (ALBANY, UNITED STATES) |
| Trial ID | NIH-11083091 on ClinicalTrials.gov |
What this research studies
This research focuses on creating RNA aptamers that can inhibit AMPA receptors, which are implicated in the progression of amyotrophic lateral sclerosis (ALS). The researchers will test the safety and effectiveness of these aptamers in a mouse model, aiming to find a new drug candidate that could alter the course of ALS. By using a novel in vitro evolution approach, they have identified potent RNA aptamers that are more selective and water-soluble than traditional small-molecule drugs, potentially leading to fewer side effects. The ultimate goal is to provide a more effective treatment option for ALS patients.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with amyotrophic lateral sclerosis (ALS).
Not a fit: Patients with other neurodegenerative disorders unrelated to ALS may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a new drug that significantly improves treatment options for ALS patients.
How similar studies have performed: While the use of RNA aptamers is a relatively novel approach, similar strategies targeting receptor inhibition have shown promise in other conditions.
Where this research is happening
ALBANY, UNITED STATES
- STATE UNIVERSITY OF NEW YORK AT ALBANY — ALBANY, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: NIU, LI — STATE UNIVERSITY OF NEW YORK AT ALBANY
- Study coordinator: NIU, LI
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