Modulation of protein folding pathways: a new platform technology for molecular medicine. Misfolding of proteins is becoming recognised as a major cause of inherited disease. We propose to develop a chemical agent that will optimise the folding of alpha1-antitrypsin (AAT), misfolding of which gives rise to inheritable liver and lung disease. This agent will have potential application as a therapy for sufferers of AAT-misfolding disease and for improving the yield of AAT purified from human plasm ....Modulation of protein folding pathways: a new platform technology for molecular medicine. Misfolding of proteins is becoming recognised as a major cause of inherited disease. We propose to develop a chemical agent that will optimise the folding of alpha1-antitrypsin (AAT), misfolding of which gives rise to inheritable liver and lung disease. This agent will have potential application as a therapy for sufferers of AAT-misfolding disease and for improving the yield of AAT purified from human plasma, which is the current agent used to treat patients with AAT-misfolding disease.Read moreRead less