Saturday, December 14, 2013

Unfolding a potential cure for disease

Researchers at Oregon Health and Science University have discovered that a misfolded protein could hold the key to unlocking cures for a wide variety of diseases.  Using small molecules, the researchers found, in mice, that the midfolded protein can be unfolded and correctly re-structured, thus allowing it to function properly.  From a press release at OHSU:
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.
Kudos to the OHSU for this potentially huge medical discovery!