Design and redesign journey of a drug for transthyretin amyloidosis

作     者：Francisca Pinheiro Salvador Ventura Francisca Pinheiro;Salvador Ventura

作者机构：Institut de Biotecnologia i BiomedicinaDepartament de Bioquímica i Biologia MolecularUniversitat Autònoma de BarcelonaBellaterraSpain 

出 版 物：《Neural Regeneration Research》 (中国神经再生研究（英文版）)

年 卷 期：2025年第20卷第4期

页      面：1096-1097页

核心收录：

学科分类：1007[医学-药学(可授医学、理学学位)] 10[医学] 

基　　金：funded by the Spanish Ministry of Science and Innovation(PDC2021-120914-I00) the Universitat Autònoma de Barcelona(PROOF OF CONCEPT 2020) ICREA,ICREA-Academia 2015 and 2020(to SV) 

主　　题：amyloid aggregation senile 

摘      要：The misfolding and subsequent aggregation of proteins into amyloid fibrils underlie the onset of a variety of human disorders collectively known as ***(TTR)is one of the30 amyloidogenic proteins identified to date and is associated with a group of highly debilitating and life-threatening disorders called TTR amyloidosis(ATTR).ATTR comprises senile systemic amyloidosis,which is linked to wild-type(WT)TTR aggregation,and hereditary ATTR,a dominantly inherited disorder caused by the deposition of one of over 130 TTR genetic *** systemic amyloidosis is a prevalent age-related amyloidosis,affecting up to 25%of the population over 80 years of age,and is characterized by the build-up of TTR fibrils in the *** hereditary ATTR,the clinical presentation is highly heterogeneous,primarily affecting the peripheral nervous system(familial amyloid polyneuropathy-FAP)or the heart(familial amyloid cardiomyopathy).In rare cases,aggregation develops in the central nervous system,giving rise to a phenotype known as familial leptomeningeal amyloidosis(Carroll et al.,2022).