Hereditary transthyretin amyloidosis: molecular basis and therapeutical strategies.

Abstract excerpt

Transthyretin (TTR) is a transport protein for thyroid hormones and vitamin A and might have an important role in the nervous system. However, TTR can undergo a conformational change and form amyloid fibrils, in both acquired and hereditary forms of systemic amyloidosis. More than 80 TTR mutations have been associated with autosomal dominant amyloidosis, usually presenting with peripheral and autonomic neuropathy...

Topics

• Amyloid
• Amyloidosis, Familial
• Cell Death
• Dimerization
• Drug Therapy
• Genetic Predisposition to Disease
• Genetic Therapy
• Humans
• Liver Transplantation
• Models, Molecular
• Mutation
• Phenotype
• Prealbumin
• Protein Conformation