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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 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.

Titel: Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis
Verfasser: Niemietz, Christoph
Chandhok, Gursimran GND
Schmidt, Hartmut GND
Organisation: FB 05: Medizinische Fakultät
Dokumenttyp: Artikel
Medientyp: Text
Erscheinungsdatum: 30.09.2015
Publikation in MIAMI: 30.11.2015
Datum der letzten Änderung: 16.09.2016
Quelle: Molecules 20 (2015) 10, 17944-17975
Schlagworte: transthyretin; familial amyloid polyneuropathy; antisense oligonucleotide; small-interfering RNA; liver
Fachgebiete: Medizin und Gesundheit
Lizenz: CC BY 4.0
Sprache: Englisch
Anmerkungen: Finanziert durch den Open-Access-Publikationsfonds 2015/2016 der Westfälischen Wilhelms-Universität Münster (WWU Münster).
Format: PDF-Dokument
URN: urn:nbn:de:hbz:6-47269568676
Permalink: http://nbn-resolving.de/urn:nbn:de:hbz:6-47269568676
DOI: 10.3390/molecules201017944
ISSN: 1420-3049
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