ReviewMolecules (Basel, Switzerland)2015
Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis.
Review in Molecules (Basel, Switzerland), 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
13 citing papers in PubMed, 31 citations in OpenAlex.
- Amyloidogenic phenotypical variation affects post-transplant outcome of hereditary transthyretin amyloidosis: a retrospective study.eGastroenterology · 2026Article
- Chemistry, structure and function of approved oligonucleotide therapeutics.Nucleic acids research · 2023Review
- Liver-directed drugs for transthyretin-mediated amyloidosis.Journal of the peripheral nervous system : JPNS · 2022Review
- Article
- Review on the Structures and Activities of Transthyretin Amyloidogenesis Inhibitors.Drug design, development and therapy · 2020Review
- Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis.Drug design, development and therapy · 2019Review
- Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs.Nucleic acids research · 2018Review
- Corino de Andrade disease: mechanisms and impact on reproduction.JBRA assisted reproduction · 2017Review
- Addressing Common Questions Encountered in the Diagnosis and Management of Cardiac Amyloidosis.Circulation · 2017Review
- A Peptide-Fc Opsonin with Pan-Amyloid Reactivity.Frontiers in immunology · 2017Article
- Gene-targeting pharmaceuticals for single-gene disorders.Human molecular genetics · 2016Review
- Evaluation of Therapeutic Oligonucleotides for Familial Amyloid Polyneuropathy in Patient-Derived Hepatocyte-Like Cells.PloS one · 2016Article
- Bolaamphiphile-based nanocomplex delivery of phosphorothioate gapmer antisense oligonucleotides as a treatment for Clostridium difficile.International journal of nanomedicine · 2016Article
Corrections and comments
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.