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Molecules 2015, 20(10), 17944-17975; doi:10.3390/molecules201017944

Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis

Klinik für Transplantationsmedizin, Universitätsklinikum Münster, Albert-Schweitzer-Campus 1, Gebäude A14, D-48149 Münster, Germany
These authors contributed equally to this work.
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Author to whom correspondence should be addressed.
Academic Editor: Ramon Eritja
Received: 13 July 2015 / Revised: 23 September 2015 / Accepted: 23 September 2015 / Published: 30 September 2015
(This article belongs to the Special Issue Frontiers in Nucleic Acid Chemistry)
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Abstract

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 linked to the establishment of extracellular protein deposition in various tissues, including the heart and the peripheral nervous system. Recent progress in the chemistry and formulation of antisense (ASO) and small interfering RNA (siRNA) designed for a knockdown of TTR mRNA in the liver has allowed to address the issue of gene-specific molecular therapy in a clinical setting of FAP. The two therapeutic oligonucleotides bind to RNA in a sequence specific manner but exploit different mechanisms. Here we describe major developments that have led to the advent of therapeutic oligonucleotides for treatment of TTR-related disease. View Full-Text
Keywords: transthyretin; familial amyloid polyneuropathy; antisense oligonucleotide; small-interfering RNA; liver transthyretin; familial amyloid polyneuropathy; antisense oligonucleotide; small-interfering RNA; liver
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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Niemietz, C.; Chandhok, G.; Schmidt, H. Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis. Molecules 2015, 20, 17944-17975.

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