BACKGROUND
Protein misfolding is thought to be the cause of many degenerative and neurodegenerative diseases such as Huntington’s disease, Parkinson’s, Alzheimer’s, and cystic fibrosis. Oftentimes the result of this misfolding is aggregations of misfolded proteins. There are some therapies to try and treat misfolded proteins or aggregates, but they heavily depend on unfolding the misfolded proteins to allow the proteins to refold themselves properly. This relies on the assumption that proper folding will be the most thermodynamically favored and will not result in any metastable configurations. Unfortunately, this approach has shown limited success, highlighting a crucial need to enable proper protein folding as an attempt to treat degenerative diseases.
SUMMARY OF TECHNOLOGY
Researchers at OSU have developed a novel strategy for developing therapeutic peptides addressing human genetic diseases that involve protein misfolding and/or aggregation defects of the protein at the basis of the genetic disease. These peptides help to correctly fold proteins that are incorrectly folded and are supported by experimental evidence as well as computational demonstrations in a model protein system. This offers an advantage over current approaches to protein misfolding therapies, which focus on unfolding the misfolded state. Instead, these peptides accelerate the correct folding of the proteins by affecting the nonequilibrium kinetic partitioning of the protein to favor correct folding. These results provide a ready-to-go strategy to develop therapeutic peptides for a range of human genetic diseases for which the three-dimensional structure of the affected protein in its native confirmation is known.
POTENTIAL AREAS OF APPLICATION
MAIN ADVANTAGES
STAGE OF DEVELOPMENT
This technology is currently available as a working model.