Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis
Published 2015 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis
Authors
Keywords
-
Journal
MOLECULES
Volume 20, Issue 10, Pages 17944-17975
Publisher
MDPI AG
Online
2015-10-01
DOI
10.3390/molecules201017944
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- Mipomersen preferentially reduces small low-density lipoprotein particle number in patients with hypercholesterolemia
- (2015) Raul D. Santos et al. Journal of Clinical Lipidology
- Going beyond the liver: Progress and challenges of targeted delivery of siRNA therapeutics
- (2015) Cornelia Lorenzer et al. JOURNAL OF CONTROLLED RELEASE
- Genetics of liver disease: From pathophysiology to clinical practice
- (2015) Tom H. Karlsen et al. JOURNAL OF HEPATOLOGY
- Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study
- (2015) Ole B Suhr et al. Orphanet Journal of Rare Diseases
- Chimeric Antisense Oligonucleotide Conjugated to α-Tocopherol
- (2015) Tomoko Nishina et al. Molecular Therapy-Nucleic Acids
- Repurposing Diflunisal for Familial Amyloid Polyneuropathy
- (2015) John L. Berk et al. JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
- Genetic variation in the non-coding genome: Involvement of micro-RNAs and long non-coding RNAs in disease
- (2014) Barbara Hrdlickova et al. BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE
- Transthyretin Antisense Oligonucleotides Lower Circulating RBP4 Levels and Improve Insulin Sensitivity in Obese Mice
- (2014) Laura Zemany et al. DIABETES
- Development of lipid nanoparticle formulations of siRNA for hepatocyte gene silencing following subcutaneous administration
- (2014) Sam Chen et al. JOURNAL OF CONTROLLED RELEASE
- Atomistic Investigation of the Effect of Incremental Modification of Deoxyribose Sugars by Locked Nucleic Acid (β-d-LNA and α-l-LNA) Moieties on the Structures and Thermodynamics of DNA–RNA Hybrid Duplexes
- (2014) Gorle Suresh et al. JOURNAL OF PHYSICAL CHEMISTRY B
- Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing
- (2014) Jayaprakash K. Nair et al. JOURNAL OF THE AMERICAN CHEMICAL SOCIETY
- Effective Dystrophin Restoration by a Novel Muscle-Homing Peptide–Morpholino Conjugate in Dystrophin-Deficient mdx Mice
- (2014) Xianjun Gao et al. MOLECULAR THERAPY
- Locked nucleic acid oligomers as handles for single molecule manipulation
- (2014) John P. Berezney et al. NUCLEIC ACIDS RESEARCH
- Targeted delivery of antisense oligonucleotides to hepatocytes using triantennaryN-acetyl galactosamine improves potency 10-fold in mice
- (2014) Thazha P. Prakash et al. NUCLEIC ACIDS RESEARCH
- Antisense Oligonucleotide Therapies
- (2014) Kendall S. Frazier TOXICOLOGIC PATHOLOGY
- Antisense Mediated Splicing Modulation For Inherited Metabolic Diseases: Challenges for Delivery
- (2014) Belen Pérez et al. Nucleic Acid Therapeutics
- Evaluation of Multiple-Turnover Capability of Locked Nucleic Acid Antisense Oligonucleotides in Cell-Free RNase H-Mediated Antisense Reaction and in Mice
- (2014) Tsuyoshi Yamamoto et al. Nucleic Acid Therapeutics
- Changes in Mipomersen Dosing Regimen Provide Similar Exposure With Improved Tolerability in Randomized Placebo‐Controlled Study of Healthy Volunteers
- (2014) JoAnn D. Flaim et al. Journal of the American Heart Association
- Shielding of Lipid Nanoparticles for siRNA Delivery: Impact on Physicochemical Properties, Cytokine Induction, and Efficacy
- (2014) Varun Kumar et al. Molecular Therapy-Nucleic Acids
- De-repressing LncRNA-Targeted Genes to Upregulate Gene Expression: Focus on Small Molecule Therapeutics
- (2014) Roya Pedram Fatemi et al. Molecular Therapy-Nucleic Acids
- Lipid Nanoparticles Improve Activity of Single-Stranded siRNA and Gapmer Antisense Oligonucleotides in Animals
- (2013) Thazha P. Prakash et al. ACS Chemical Biology
- Molecular Mechanisms of RNA Interference
- (2013) Ross C. Wilson et al. Annual Review of Biophysics
- Covalent conjugation of oligonucleotides with cell-targeting ligands
- (2013) Md. Rowshon Alam et al. BIOORGANIC & MEDICINAL CHEMISTRY
- Is mipomersen ready for clinical implementation? A transatlantic dilemma
- (2013) Barbara Sjouke et al. CURRENT OPINION IN LIPIDOLOGY
- Mipomersen Sodium: First Global Approval
- (2013) Philip Hair et al. DRUGS
- Long-term efficacy and safety of mipomersen in patients with familial hypercholesterolaemia: 2-year interim results of an open-label extension
- (2013) Raul D. Santos et al. EUROPEAN HEART JOURNAL
- Is there a future for cell-penetrating peptides in oligonucleotide delivery?
- (2013) Soo Hyeon Lee et al. EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS
- Liver as a target for oligonucleotide therapeutics
- (2013) Alfica Sehgal et al. JOURNAL OF HEPATOLOGY
- Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy
- (2013) Teresa Coelho et al. JOURNAL OF NEUROLOGY
- Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial
- (2013) Kevin Fitzgerald et al. LANCET
- Hepatocyte-targeted RNAi Therapeutics for the Treatment of Chronic Hepatitis B Virus Infection
- (2013) Christine I Wooddell et al. MOLECULAR THERAPY
- Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis
- (2013) Teresa Coelho et al. NEW ENGLAND JOURNAL OF MEDICINE
- Guideline of transthyretin-related hereditary amyloidosis for clinicians
- (2013) Yukio Ando et al. Orphanet Journal of Rare Diseases
- Antisense Technology: An Emerging Platform for Cardiovascular Disease Therapeutics
- (2013) Richard G. Lee et al. Journal of Cardiovascular Translational Research
- Effects of Tafamidis on Transthyretin Stabilization and Clinical Outcomes in Patients with Non-Val30Met Transthyretin Amyloidosis
- (2013) Giampaolo Merlini et al. Journal of Cardiovascular Translational Research
- Hepatic RNA interference: delivery by synthetic vectors
- (2013) Matthew T. Haynes et al. Drug Delivery and Translational Research
- Lipid nanoparticle delivery systems for siRNA-based therapeutics
- (2013) C. Wan et al. Drug Delivery and Translational Research
- Genotype – phenotype correlation in FAP
- (2012) Steven R. Zeldenrust AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS
- Clinical development of an antisense therapy for the treatment of transthyretin-associated polyneuropathy
- (2012) Elizabeth J. Ackermann et al. AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS
- Transthyretin (TTR) Cardiac Amyloidosis
- (2012) Frederick L. Ruberg et al. CIRCULATION
- THAOS – The Transthyretin Amyloidosis Outcomes Survey: initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis
- (2012) Teresa Coelho et al. CURRENT MEDICAL RESEARCH AND OPINION
- Therapeutic Potential of Mipomersen in the Management of Familial Hypercholesterolaemia
- (2012) Carmen Gelsinger et al. DRUGS
- Oral delivery of small RNA and DNA
- (2012) Diane C. Forbes et al. JOURNAL OF CONTROLLED RELEASE
- Inhibition of natural antisense transcripts in vivo results in gene-specific transcriptional upregulation
- (2012) Farzaneh Modarresi et al. NATURE BIOTECHNOLOGY
- RNA therapeutics: beyond RNA interference and antisense oligonucleotides
- (2012) Ryszard Kole et al. NATURE REVIEWS DRUG DISCOVERY
- Tafamidis for transthyretin familial amyloid polyneuropathy: A randomized, controlled trial
- (2012) T. Coelho et al. NEUROLOGY
- Target mRNA inhibition by oligonucleotide drugs in man
- (2012) H. L. Lightfoot et al. NUCLEIC ACIDS RESEARCH
- Randomized, Placebo-Controlled Trial of Mipomersen in Patients with Severe Hypercholesterolemia Receiving Maximally Tolerated Lipid-Lowering Therapy
- (2012) Mary P. McGowan et al. PLoS One
- Defining the Diagnosis in Echocardiographically Suspected Senile Systemic Amyloidosis
- (2012) Candida Cristina Quarta et al. JACC-Cardiovascular Imaging
- Antisense oligonucleotide therapy for TTR amyloidosis
- (2011) M. D. Benson et al. AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS
- Modeling RNA interference in mammalian cells
- (2011) Giulia Cuccato et al. BMC Systems Biology
- Phosphorothioate Nucleotides and Oligonucleotides - Recent Progress in Synthesis and Application
- (2011) Piotr Guga et al. CHEMISTRY & BIODIVERSITY
- Cellular delivery of siRNA and antisense oligonucleotides via receptor-mediated endocytosis
- (2011) Xin Ming Expert Opinion on Drug Delivery
- Hepatic cell-to-cell transmission of small silencing RNA can extend the therapeutic reach of RNA interference (RNAi)
- (2011) Qiuwei Pan et al. GUT
- Synthesis and in vitro inhibition properties of siRNA conjugates carrying glucose and galactose with different presentations
- (2011) Anna Aviñó et al. MOLECULAR DIVERSITY
- Is RNAi Dead?
- (2011) Arthur M Krieg MOLECULAR THERAPY
- Chromatin-associated RNA interference components contribute to transcriptional regulation in Drosophila
- (2011) Filippo M. Cernilogar et al. NATURE
- Current prospects for RNA interference-based therapies
- (2011) Beverly L. Davidson et al. NATURE REVIEWS GENETICS
- Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy
- (2011) Nathalie M. Goemans et al. NEW ENGLAND JOURNAL OF MEDICINE
- Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes
- (2011) Erich Koller et al. NUCLEIC ACIDS RESEARCH
- Direct Translocation as Major Cellular Uptake for CADY Self-Assembling Peptide-Based Nanoparticles
- (2011) Anna Rydström et al. PLoS One
- Suppression of choroid plexus transthyretin levels by antisense oligonucleotide treatment
- (2010) Merrill D. Benson et al. AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS
- RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform
- (2010) C. Frank Bennett et al. Annual Review of Pharmacology and Toxicology
- Insights into RNA/DNA hybrid recognition and processing by RNase H from the crystal structure of a non-specific enzyme-dsDNA complex
- (2010) Pradeep S. Pallan et al. CELL CYCLE
- Breaking down the barriers: siRNA delivery and endosome escape
- (2010) M. Dominska et al. JOURNAL OF CELL SCIENCE
- Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial
- (2010) Frederick J Raal et al. LANCET
- Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model
- (2010) Hans Heemskerk et al. MOLECULAR THERAPY
- Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles
- (2010) Mark E. Davis et al. NATURE
- Transthyretin-related amyloidoses and the heart: a clinical overview
- (2010) Claudio Rapezzi et al. Nature Reviews Cardiology
- Intracellular Traffic of Oligodeoxynucleotides In and Out of the Nucleus: Effect of Exportins and DNA Structure
- (2010) Stephen J. Forsha et al. OLIGONUCLEOTIDES
- Polerovirus protein P0 prevents the assembly of small RNA-containing RISC complexes and leads to degradation of ARGONAUTE1
- (2010) Tibor Csorba et al. PLANT JOURNAL
- Transthyretin-related familial amyloidotic polyneuropathy—Progress in Kumamoto, Japan (1967–2010)—
- (2010) Shukuro ARAKI et al. PROCEEDINGS OF THE JAPAN ACADEMY SERIES B-PHYSICAL AND BIOLOGICAL SCIENCES
- Effect of dose and plasma concentration on liver uptake and pharmacologic activity of a 2′-methoxyethyl modified chimeric antisense oligonucleotide targeting PTEN
- (2009) Richard S. Geary et al. BIOCHEMICAL PHARMACOLOGY
- Antisense oligonucleotide pharmacokinetics and metabolism
- (2009) Richard S Geary Expert Opinion on Drug Metabolism & Toxicology
- Ribonuclease H: the enzymes in eukaryotes
- (2009) Susana M. Cerritelli et al. FEBS Journal
- Non-coding RNAs: regulators of disease
- (2009) Ryan J Taft et al. JOURNAL OF PATHOLOGY
- Liver transplantation for familial amyloidotic polyneuropathy: Impact on Swedish patients' survival
- (2009) Sadahisa Okamoto et al. LIVER TRANSPLANTATION
- Biological Barriers to Therapy with Antisense and siRNA Oligonucleotides
- (2009) R. Juliano et al. MOLECULAR PHARMACEUTICS
- A large-scale chemical modification screen identifies design rules to generate siRNAs with high activity, high stability and low toxicity
- (2009) Jesper B. Bramsen et al. NUCLEIC ACIDS RESEARCH
- Increased RNAi is related to intracellular release of siRNA via a covalently attached signal peptide
- (2009) A. Detzer et al. RNA
- Senile systemic amyloidosis affects 25% of the very aged and associates with genetic variation inalpha2‐macroglobulinandtau: A population‐based autopsy study
- (2008) Maarit Tanskanen et al. ANNALS OF MEDICINE
- Factors Determining the Efficacy of Nuclear Delivery of Antisense Oligonucleotides by Gold Nanoparticles
- (2008) Yanli Liu et al. BIOCONJUGATE CHEMISTRY
- Efficient In Vivo Delivery of siRNA to the Liver by Conjugation of α-Tocopherol
- (2008) Kazutaka Nishina et al. MOLECULAR THERAPY
- Delivery of proteins and nucleic acids using a non-covalent peptide-based strategy
- (2007) Sébastien Deshayes et al. ADVANCED DRUG DELIVERY REVIEWS
Find Funding. Review Successful Grants.
Explore over 25,000 new funding opportunities and over 6,000,000 successful grants.
ExploreBecome a Peeref-certified reviewer
The Peeref Institute provides free reviewer training that teaches the core competencies of the academic peer review process.
Get Started