How the huntingtin protein misfolds and ways proteins might stop it
Molecular Mechanism of Huntingtin Misfolding and its Inhibition by Designed and Cellular Proteins
Researchers are mapping the 3D shapes of misfolding huntingtin and testing designed and natural proteins to block the harmful clumps that drive Huntington's disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Southern California NIH-funded |
| Lab location | 1 site (Los Angeles, UNITED STATES) |
| Project ID | NIH-11174241 on NIH RePORTER |
What this research studies
From a patient's perspective, scientists are isolating the different shapes that the huntingtin protein takes as it misfolds and clumps together in Huntington's disease. They will use high-resolution tools like NMR, cryo-EM, and EPR alongside cell-based toxicity tests to see which shapes are most harmful. The team is also testing designed proteins and natural chaperone proteins to see if they can bind to specific misfolded forms and stop aggregation. These laboratory findings aim to point to strategies that could be developed into future treatments.
Who could benefit from this research
Good fit: People living with Huntington's disease or those known to carry the HD genetic expansion would be most directly relevant to the goals and future applications of this research.
Not a fit: People without Huntington's disease or those with unrelated conditions are unlikely to see direct benefits from this project in the near term.
Why it matters
Potential benefit: If successful, this work could identify targets and protein-based strategies to prevent huntingtin aggregation and slow disease progression.
How similar studies have performed: Prior lab studies have characterized misfolded huntingtin forms and shown that chaperones or designed molecules can reduce aggregation in cells and animal models, but converting those findings into proven patient therapies remains unachieved.
Where this research is happening
Los Angeles, UNITED STATES
- University of Southern California — Los Angeles, United States (Active)
Researchers
- Principal investigator: Langen, Ralf — University of Southern California
- Study coordinator: Langen, Ralf
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.