Proteins adopt their functional three-dimensional structure by the folding of a linear chain of amino acids. Gene mutation can cause this folding process to go awry, resulting in "misfolded" proteins that are inactive or, in worse cases, exhibit modified or toxic functionality. This is the cause of a wide range of diseases, but researchers have developed a technique that fixes these misfolded proteins, allowing them to perform their intended function, thereby providing a potential cure for a number of diseases.
Up until relatively recently, scientists believed that misfolded proteins that were inactive were intrinsically non-functional. However, it was discovered that their inactivity was due to the cell's quality control system misrouting them within the cell. Drugs called "pharmacoperones," which get their name from their ability to act as so-called "protein chaperones," have the ability to enter cells and fix the misfolded proteins so they can be routed correctly, thus restoring their functionality.