Next Article in Journal
Comparative Studies on Phenolic Composition, Antioxidant, Wound Healing and Cytotoxic Activities of Selected Achillea L. Species Growing in Turkey
Next Article in Special Issue
Synthesis, Hydrolysis, and Protonation-Promoted Intramolecular Reductive Breakdown of Potential NRTIs: Stavudine α-P-Borano-γ-P-N-l-tryptophanyltriphosphates
Previous Article in Journal
Ionic Liquid-Based Ultrasonic-Assisted Extraction of Secoisolariciresinol Diglucoside from Flaxseed (Linum usitatissimum L.) with Further Purification by an Aqueous Two-Phase System
Previous Article in Special Issue
Comparative Incorporation of PNA into DNA Nanostructures
Open AccessReview

Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis

Klinik für Transplantationsmedizin, Universitätsklinikum Münster, Albert-Schweitzer-Campus 1, Gebäude A14, D-48149 Münster, Germany
*
Author to whom correspondence should be addressed.
These authors contributed equally to this work.
Academic Editor: Ramon Eritja
Molecules 2015, 20(10), 17944-17975; https://doi.org/10.3390/molecules201017944
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)
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
Show Figures

Figure 1

MDPI and ACS Style

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

Show more citation formats Show less citations formats

Article Access Map by Country/Region

1
Only visits after 24 November 2015 are recorded.
Back to TopTop