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Article

Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis.

Molecules (Basel, Switzerland) - 30 Sept 2015

Niemietz Christoph, Chandhok Gursimran, Schmidt Hartmut

Abstract excerpt

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...

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Topics

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Share to Amyloid Neuropathies, Familial
Share to Drug Evaluation, Preclinical
Share to Gene Silencing

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Genetic Therapy

Share to Liver Diseases

Share to Oligonucleotides

Share to Oligonucleotides, Antisense

Share to Prealbumin

Source

Open in PubMed Open full text

Identifiers

PMID: 26437390
DOI: 10.3390/molecules201017944
PMCID: PMC6332041

Genes

TTR
FAP

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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