Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis

The liver has become an increasingly interesting target for oligonucleotide therapy. Mutations of the gene encoding transthyretin (TTR), expressed in vast amounts by the liver, result in a complex degenerative disease, termed familial amyloid polyneuropathy (FAP). Misfolded variants of TTR are linke...

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Bibliographic Details
Main Authors: Christoph Niemietz, Gursimran Chandhok, Hartmut Schmidt
Format: Article
Language:English
Published: MDPI AG 2015-09-01
Series:Molecules
Subjects:
Online Access:http://www.mdpi.com/1420-3049/20/10/17944