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...
Main Authors: | , , |
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Format: | Article |
Language: | English |
Published: |
MDPI AG
2015-09-01
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Series: | Molecules |
Subjects: | |
Online Access: | http://www.mdpi.com/1420-3049/20/10/17944 |