” A team led by a longtime Oregon Health & Science University researcher has demonstrated in mice what could be a revolutionary new technique to cure a wide range of human diseases — from cystic fibrosis to cataracts to Alzheimer’s disease — that are caused by “misfolded” protein molecules.
Misfolded protein molecules, caused by gene mutation, are capable of maintaining their function but are misrouted within the cell and can’t work normally, thus causing disease. The OHSU team discovered a way to use small molecules that enter cells, fix the misfolded proteins and allow the proteins to move to the correct place and function normally again. “
Continue reading OHSU researchers develop new drug approach that could lead to cures for wide range of diseases,
Oregon Health and Science University, 09 Dec 2013
- Restoration of testis function in hypogonadotropic hypogonadal mice harboring a misfolded GnRHR mutant by pharmacoperone drug therapy, PNAS, 10 Sep 2013
- Supporting Information, Janovick et al. 10.1073/pnas.1315194110