Project title: “TTR amyloid fiber formation in vivo – looking beyond genetic factors”
Funding: 50 000 USD
Summary:
Familial amyloidotic polyneuropathy is a crippling and fatal disease affecting an increasing number of people worldwide. The only currently available therapeutic option is liver transplantation, an invasive approach not exempt of complications and affecting patient’s quality of life. This project aims to discover new therapeutic opportunities by exploring nongenetic factors affecting these patients. We intend to explore protein modification and protein folding regulatory networks that may be affected in this pathology.