Patisiran in hATTR Amyloidosis: Six-Month Latency Period before Efficacy

Patisiran in hATTR Amyloidosis: Six-Month Latency Period before Efficacy

Hereditary amyloidosis associated with mutations in the transthyretin gene (hATTR) is a progressive devastating disease, with a fatal outcome occurring within 10years after onset. In recent years, TTR gene silencing therapy appeared as a promising therapeutic strategy, showing evidence that disease...

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Bibliographic Details
Main Authors: Luca Gentile, Massimo Russo, Marco Luigetti, Giulia Bisogni, Andrea Di Paolantonio, Angela Romano, Valeria Guglielmino, Ilenia Arimatea, Mario Sabatelli, Antonio Toscano, Giuseppe Vita, Anna Mazzeo
Format: Article
Language:English
Published: MDPI AG 2021-04-01
Series:Brain Sciences
Subjects:
hATTR
amyloidosis
TTR silencers
gene therapy
Online Access:https://www.mdpi.com/2076-3425/11/4/515

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