How the brain transporter NaDC3 moves key molecules and can be blocked
Transport Cycle and Inhibition of Human Na+-dicarboxylate Cotransporter NaDC3
['FUNDING_R01'] · NEW YORK UNIVERSITY SCHOOL OF MEDICINE · NIH-11259452
Researchers are looking at how the NaDC3 protein in the brain moves molecules like N-acetyl-aspartate and whether blocking it could help people with Canavan disease.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | NEW YORK UNIVERSITY SCHOOL OF MEDICINE (nih funded) |
| Locations | 1 site (NEW YORK, UNITED STATES) |
| Trial ID | NIH-11259452 on ClinicalTrials.gov |
What this research studies
This project aims to map the shape and movement of the human NaDC3 transporter as it carries dicarboxylates into brain cells. The team will test how small molecules bind and block the transporter and will observe the protein switching between outward- and inward-facing states. Work uses purified human protein, structural and biochemical methods, and inhibitor testing focused on metabolites such as N-acetyl-aspartate that are linked to Canavan disease. Findings could guide the design of drugs to limit harmful metabolite buildup in the brain.
Who could benefit from this research
Good fit: People with Canavan disease or related disorders that affect N-acetyl-aspartate metabolism would be the most relevant candidates to follow this research or participate in related sample/donor efforts.
Not a fit: People with neurological conditions that do not involve N-acetyl-aspartate or the NaDC3 transporter are unlikely to receive direct benefit from this project.
Why it matters
Potential benefit: If successful, the work could point to drugs that reduce N-acetyl-aspartate overload and help protect white matter in people with Canavan disease.
How similar studies have performed: Some small-molecule inhibitors of NaDC3 and related transporter structures have been reported, but a full, detailed picture of NaDC3's transport cycle and inhibition is still emerging.
Where this research is happening
NEW YORK, UNITED STATES
- NEW YORK UNIVERSITY SCHOOL OF MEDICINE — NEW YORK, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: WANG, DANENG — NEW YORK UNIVERSITY SCHOOL OF MEDICINE
- Study coordinator: WANG, DANENG
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: Aspartoacylase Deficiency Disease