Targeting HCN1 channels to develop better treatments for infantile epileptic encephalopathy
Uncovering Druggable Allosteric Sites in HCN Channels
Researchers are looking for specific spots on HCN1 proteins that new medicines could target to help children with infantile epileptic encephalopathy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Weill Medical Coll of Cornell Univ NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11283943 on NIH RePORTER |
What this research studies
I have infantile epileptic encephalopathy linked to HCN1 mutations, and researchers are studying how the HCN1 protein works and where drugs might bind. They will combine patient genetic information with lab tests of normal and mutant channels and determine high-resolution structures of the channel bound to molecules that either activate or inhibit it. The team will use computer models of protein movements to connect how mutations and small molecules change channel behavior and to map promising drug-binding sites. This approach aims to create a clear plan for designing medicines that act precisely on faulty HCN1 channels.
Who could benefit from this research
Good fit: Ideal candidates would be children (and their families) with infantile epileptic encephalopathy caused by known or suspected HCN1 mutations who can share genetic information or biological samples.
Not a fit: People with epilepsy caused by other genes or non-HCN1 conditions are unlikely to receive direct benefit from these HCN1-specific discoveries.
Why it matters
Potential benefit: If successful, this work could lead to targeted medicines for children with HCN1-related infantile epileptic encephalopathy that are more effective and have fewer side effects.
How similar studies have performed: Structural and allosteric drug-design approaches have produced targeted therapies for other ion-channel disorders, but applying them specifically to HCN1 in infantile epileptic encephalopathy is largely new.
Where this research is happening
New York, United States
- Weill Medical Coll of Cornell Univ — New York, United States (Active)
Researchers
- Principal investigator: Nimigean, Crina M — Weill Medical Coll of Cornell Univ
- Study coordinator: Nimigean, Crina M
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.