This technology provides a therapeutic method for reducing the levels of toxic mutant huntingtin protein present in an individual. This is accomplished by increasing expression or activity of selected quality control proteins in cells of Huntington‘s disease gene positive patients or those manifesting the disease. The technology also reduces protein misfolding in cells, promoting normal or improved cellular protein quality control by increasing expression of thiol transferases in affected cells effective for manipulating endoplasmic reticulum (ER) and cytosolic quality control.
Huntington disease affects approximately 30,000 individuals in the United State and many more worldwide. This technology can be applied in a therapeutic setting, providing relief for those patients or potential persons affected by protein misfolding neurodegenerative diseases. Using this technology could regulate the huntingtin protein misfolds which accumulate in the brain and ultimately result in loss of brain functions.
The benefits of this technology are the potential to help those who are or may become affected by a protein misfolding neurodegenerative disease.