There’s a New Drug in Town
PROTEINS are biological molecules that perform many functions in the body. Specific proteins have very specific functions depending on their molecular sequence and the resulting 3-Dimensional “folded” structures. Check out how a protein folds in this video!
Many diseases are a result of genetic mutations that cause proteins to fold improperly. When this happens, the misfolded proteins do not pass the cellular protein production ‘quality control’ and are sequestered deep inside the cell – even if they may still be functional. This results in loss of function of the protein and eventual diseases (check out the piece on protein misfolding in the journal, Nature). An innovative treatment that has been years in the making, has recently been shown to work in animal models to restore protein function by fixing the misfolded proteins and allowing the proteins to move to the correct place and function normally again. This is done with the help of a small molecule Pharmacological Chaperone - PHARMACOPERONE, a unique class of target-specific drugs that assist in protein folding by acting as folding templates. This technique worked in mice - the next step is to see if this will work in humans!
publication in PNAS
image from Science Daily