Related references
Note: Only part of the references are listed.Mipomersen preferentially reduces small Low-density lipoprotein particle number in patients with hypercholesterolemia
Raul D. Santos et al.
JOURNAL OF CLINICAL LIPIDOLOGY (2015)
Going beyond the liver: Progress and challenges of targeted delivery of siRNA therapeutics
Cornelia Lorenzer et al.
JOURNAL OF CONTROLLED RELEASE (2015)
Genetics of liver disease: From pathophysiology to clinical practice
Tom H. Karlsen et al.
JOURNAL OF HEPATOLOGY (2015)
Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study
Ole B. Suhr et al.
ORPHANET JOURNAL OF RARE DISEASES (2015)
Chimeric Antisense Oligonucleotide Conjugated to α-Tocopherol
Tomoko Nishina et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2015)
Transthyretin Antisense Oligonucleotides Lower Circulating RBP4 Levels and Improve Insulin Sensitivity in Obese Mice
Laura Zemany et al.
DIABETES (2015)
Long-term efficacy and safety of mipomersen in patients with familial hypercholesterolaemia: 2-year interim results of an open-label extension
Raul D. Santos et al.
EUROPEAN HEART JOURNAL (2015)
Antisense Oligonucleotide Therapies: The Promise and the Challenges from a Toxicologic Pathologist's Perspective
Kendall S. Frazier
TOXICOLOGIC PATHOLOGY (2015)
Genetic variation in the non-coding genome: Involvement of micro-RNAs and long non-coding RNAs in disease
Barbara Hrdlickova et al.
BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2014)
Development of lipid nanoparticle formulations of siRNA for hepatocyte gene silencing following subcutaneous administration
Sam Chen et al.
JOURNAL OF CONTROLLED RELEASE (2014)
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
Gorle Suresh et al.
JOURNAL OF PHYSICAL CHEMISTRY B (2014)
Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing
Jayaprakash K. Nair et al.
JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2014)
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
Kevin Fitzgerald et al.
LANCET (2014)
Effective Dystrophin Restoration by a Novel Muscle-Homing Peptide-Morpholino Conjugate in Dystrophin-Deficient mdx Mice
Xianjun Gao et al.
MOLECULAR THERAPY (2014)
Locked nucleic acid oligomers as handles for single molecule manipulation
John P. Berezney et al.
NUCLEIC ACIDS RESEARCH (2014)
Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice
Thazha P. Prakash et al.
NUCLEIC ACIDS RESEARCH (2014)
Antisense Mediated Splicing Modulation For Inherited Metabolic Diseases: Challenges for Delivery
Belen Perez et al.
NUCLEIC ACID THERAPEUTICS (2014)
Evaluation of Multiple-Turnover Capability of Locked Nucleic Acid Antisense Oligonucleotides in Cell-Free RNase H-Mediated Antisense Reaction and in Mice
Tsuyoshi Yamamoto et al.
NUCLEIC ACID THERAPEUTICS (2014)
Changes in Mipomersen Dosing Regimen Provide Similar Exposure With Improved Tolerability in Randomized Placebo-Controlled Study of Healthy Volunteers
JoAnn D. Flaim et al.
JOURNAL OF THE AMERICAN HEART ASSOCIATION (2014)
Shielding of Lipid Nanoparticles for siRNA Delivery: Impact on Physicochemical Properties, Cytokine Induction, and Efficacy
Varun Kumar et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2014)
De-repressing LncRNA-Targeted Genes to Upregulate Gene Expression: Focus on Small Molecule Therapeutics
Roya Pedram Fatemi et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2014)
Hepatic RNA interference: delivery by synthetic vectors
Matthew T. Haynes et al.
DRUG DELIVERY AND TRANSLATIONAL RESEARCH (2014)
Lipid nanoparticle delivery systems for siRNA-based therapeutics
C. Wan et al.
DRUG DELIVERY AND TRANSLATIONAL RESEARCH (2014)
Vitreous Amyloidosis as the Presenting Symptom of Familial Amyloid Polyneuropathy TTR Val30Met in a Portuguese Patient
Mariana Seca et al.
CASE REPORTS IN OPHTHALMOLOGY (2014)
Lipid Nanoparticles Improve Activity of Single-Stranded siRNA and Gapmer Antisense Oligonucleotides in Animals
Thazha P. Prakash et al.
ACS CHEMICAL BIOLOGY (2013)
Molecular Mechanisms of RNA Interference
Ross C. Wilson et al.
ANNUAL REVIEW OF BIOPHYSICS, VOL 42 (2013)
Covalent conjugation of oligonucleotides with cell-targeting ligands
Md. Rowshon Alam et al.
BIOORGANIC & MEDICINAL CHEMISTRY (2013)
THAOS - The Transthyretin Amyloidosis Outcomes Survey: initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis
Teresa Coelho et al.
CURRENT MEDICAL RESEARCH AND OPINION (2013)
Is mipomersen ready for clinical implementation? A transatlantic dilemma
Barbara Sjouke et al.
CURRENT OPINION IN LIPIDOLOGY (2013)
Mipomersen Sodium: First Global Approval
Philip Hair et al.
DRUGS (2013)
Is there a future for cell-penetrating peptides in oligonucleotide delivery?
Soo Hyeon Lee et al.
EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS (2013)
Repurposing Diflunisal for Familial Amyloid Polyneuropathy A Randomized Clinical Trial
John L. Berk et al.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION (2013)
Liver as a target for oligonucleotide therapeutics
Alfica Sehgal et al.
JOURNAL OF HEPATOLOGY (2013)
Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy
Teresa Coelho et al.
JOURNAL OF NEUROLOGY (2013)
Hepatocyte-targeted RNAi Therapeutics for the Treatment of Chronic Hepatitis B Virus Infection
Christine I. Wooddell et al.
MOLECULAR THERAPY (2013)
Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis
Teresa Coelho et al.
NEW ENGLAND JOURNAL OF MEDICINE (2013)
Guideline of transthyretin-related hereditary amyloidosis for clinicians
Yukio Ando et al.
ORPHANET JOURNAL OF RARE DISEASES (2013)
Antisense Technology: An Emerging Platform for Cardiovascular Disease Therapeutics
Richard G. Lee et al.
JOURNAL OF CARDIOVASCULAR TRANSLATIONAL RESEARCH (2013)
Effects of Tafamidis on Transthyretin Stabilization and Clinical Outcomes in Patients with Non-Val30Met Transthyretin Amyloidosis
Giampaolo Merlini et al.
JOURNAL OF CARDIOVASCULAR TRANSLATIONAL RESEARCH (2013)
Oligonucleotide conjugates for therapeutic applications
Johannes Winkler
THERAPEUTIC DELIVERY (2013)
Genotype - phenotype correlation in FAP
Steven R. Zeldenrust
AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS (2012)
Clinical development of an antisense therapy for the treatment of transthyretin-associated polyneuropathy
Elizabeth J. Ackermann et al.
AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS (2012)
Transthyretin (TTR) Cardiac Amyloidosis
Frederick L. Ruberg et al.
CIRCULATION (2012)
Therapeutic Potential of Mipomersen in the Management of Familial Hypercholesterolaemia
Carmen Gelsinger et al.
DRUGS (2012)
Hepatic cell-to-cell transmission of small silencing RNA can extend the therapeutic reach of RNA interference (RNAi)
Qiuwei Pan et al.
GUT (2012)
Oral delivery of small RNA and DNA
Diane C. Forbes et al.
JOURNAL OF CONTROLLED RELEASE (2012)
Inhibition of natural antisense transcripts in vivo results in gene-specific transcriptional upregulation
Farzaneh Modarresi et al.
NATURE BIOTECHNOLOGY (2012)
RNA therapeutics: beyond RNA interference and antisense oligonucleotides
Ryszard Kole et al.
NATURE REVIEWS DRUG DISCOVERY (2012)
Tafamidis for transthyretin familial amyloid polyneuropathy A randomized, controlled trial
Teresa Coelho et al.
NEUROLOGY (2012)
Target mRNA inhibition by oligonucleotide drugs in man
Helen L. Lightfoot et al.
NUCLEIC ACIDS RESEARCH (2012)
Randomized, Placebo-Controlled Trial of Mipomersen in Patients with Severe Hypercholesterolemia Receiving Maximally Tolerated Lipid-Lowering Therapy
Mary P. McGowan et al.
PLOS ONE (2012)
Defining the Diagnosis in Echocardiographically Suspected Senile Systemic Amyloidosis
Candida Cristina Quarta et al.
JACC-CARDIOVASCULAR IMAGING (2012)
Antisense oligonucleotide therapy for TTR amyloidosis
M. D. Benson et al.
AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS (2011)
Modeling RNA interference in mammalian cells
Giulia Cuccato et al.
BMC SYSTEMS BIOLOGY (2011)
Phosphorothioate Nucleotides and Oligonucleotides - Recent Progress in Synthesis and Application
Piotr Guga et al.
CHEMISTRY & BIODIVERSITY (2011)
Cellular delivery of siRNA and antisense oligonucleotides via receptor-mediated endocytosis
Xin Ming
EXPERT OPINION ON DRUG DELIVERY (2011)
Synthesis and in vitro inhibition properties of siRNA conjugates carrying glucose and galactose with different presentations
Anna Avino et al.
MOLECULAR DIVERSITY (2011)
Is RNAi Dead?
Arthur M. Krieg
MOLECULAR THERAPY (2011)
Chromatin-associated RNA interference components contribute to transcriptional regulation in Drosophila
Filippo M. Cernilogar et al.
NATURE (2011)
Current prospects for RNA interference-based therapies
Beverly L. Davidson et al.
NATURE REVIEWS GENETICS (2011)
Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy
Nathalie M. Goemans et al.
NEW ENGLAND JOURNAL OF MEDICINE (2011)
Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes
Erich Koller et al.
NUCLEIC ACIDS RESEARCH (2011)
Direct Translocation as Major Cellular Uptake for CADY Self-Assembling Peptide-Based Nanoparticles
Anna Rydstroem et al.
PLOS ONE (2011)
Suppression of choroid plexus transthyretin levels by antisense oligonucleotide treatment
Merrill D. Benson et al.
AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS (2010)
RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform
C. Frank Bennett et al.
ANNUAL REVIEW OF PHARMACOLOGY AND TOXICOLOGY (2010)
Breaking down the barriers: siRNA delivery and endosome escape
Monika Dominska et al.
JOURNAL OF CELL SCIENCE (2010)
Non-coding RNAs: regulators of disease
Ryan J. Taft et al.
JOURNAL OF PATHOLOGY (2010)
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
Frederick J. Raal et al.
LANCET (2010)
Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model
Hans Heemskerk et al.
MOLECULAR THERAPY (2010)
Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles
Mark E. Davis et al.
NATURE (2010)
Transthyretin-related amyloidoses and the heart: a clinical overview
Claudio Rapezzi et al.
NATURE REVIEWS CARDIOLOGY (2010)
Intracellular Traffic of Oligodeoxynucleotides In and Out of the Nucleus: Effect of Exportins and DNA Structure
Stephen J. Forsha et al.
OLIGONUCLEOTIDES (2010)
Polerovirus protein P0 prevents the assembly of small RNA-containing RISC complexes and leads to degradation of ARGONAUTE1
Tibor Csorba et al.
PLANT JOURNAL (2010)
Transthyretin-related familial amyloidotic polyneuropathy -Progress in Kumamoto, Japan (1967-2010)-
Shukuro Araki et al.
PROCEEDINGS OF THE JAPAN ACADEMY SERIES B-PHYSICAL AND BIOLOGICAL SCIENCES (2010)
Effect of dose and plasma concentration on liver uptake and pharmacologic activity of a 2′-methoxyethyl modified chimeric antisense oligonucleotide targeting PTEN
Richard S. Geary et al.
BIOCHEMICAL PHARMACOLOGY (2009)
Antisense oligonucleotide pharmacokinetics and metabolism
Richard S. Geary
EXPERT OPINION ON DRUG METABOLISM & TOXICOLOGY (2009)
Ribonuclease H: the enzymes in eukaryotes
Susana M. Cerritelli et al.
FEBS JOURNAL (2009)
Biological Barriers to Therapy with Antisense and siRNA Oligonucleotides
R. Juliano et al.
MOLECULAR PHARMACEUTICS (2009)
Liver Transplantation for Familial Amyloidotic Polyneuropathy: Impact on Swedish Patients' Survival
Sadahisa Okamoto et al.
LIVER TRANSPLANTATION (2009)
A large-scale chemical modification screen identifies design rules to generate siRNAs with high activity, high stability and low toxicity
Jesper B. Bramsen et al.
NUCLEIC ACIDS RESEARCH (2009)
Increased RNAi is related to intracellular release of siRNA via a covalently attached signal peptide
Anke Detzer et al.
RNA (2009)
Delivery of proteins and nucleic acids using a non-covalent peptide-based strategy
Sebastien Deshayes et al.
ADVANCED DRUG DELIVERY REVIEWS (2008)
Senile systemic amyloidosis affects 25% of the very aged and associates with genetic variation in alpha2-macroglobulin and tau: A population-based autopsy study
Maarit Tanskanen et al.
ANNALS OF MEDICINE (2008)
Factors determining the efficacy of nuclear delivery of antisense oligonucleotides by gold nanoparticles
Yanli Liu et al.
BIOCONJUGATE CHEMISTRY (2008)
Insights into RNA/DNA hybrid recognition and processing by RNase H from the crystal structure of a non-specific enzyme-dsDNA complex
Pradeep S. Pallan et al.
CELL CYCLE (2008)
Efficient in vivo delivery of siRNA to the liver by conjugation of α-tocopherol
Kazutaka Nishina et al.
MOLECULAR THERAPY (2008)
The molecular biology and clinical features of amyloid neuropathy
Merrill D. Benson et al.
MUSCLE & NERVE (2007)
Dynamic PolyConjugates for targeted in vivo delivery of siRNA to hepatocytes
David B. Rozema et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2007)
Molecular basis for target RNA recognition and cleavage by human RISC
Stefan Ludwig Ameres et al.
CELL (2007)
Progressive wild-type transthyretin deposition after liver transplantation preferentially occurs onto myocardium in FAP patients
M. Yazaki et al.
AMERICAN JOURNAL OF TRANSPLANTATION (2007)
Strategies for silencing human disease using RNA interference
Daniel H. Kim et al.
NATURE REVIEWS GENETICS (2007)
RNA targeting with peptide conjugates of oligonucleotides, siRNA and PNA
John J. Turner et al.
BLOOD CELLS MOLECULES AND DISEASES (2007)
Determination of the hepatocellularity number for human, dog, rabbit, rat and mouse livers from protein concentration measurements
Anna-Karin Sohlenius-Sternbeck
TOXICOLOGY IN VITRO (2006)
Potent reduction of apolipoprotein B and low-density lipoprotein cholesterol by short-term administration of an antisense inhibitor of apolipoprotein B
John J. P. Kastelein et al.
CIRCULATION (2006)
Cell type-specific delivery of siRNAs with aptamer-siRNA chimeras
James O. McNamara et al.
NATURE BIOTECHNOLOGY (2006)
Position-specific chemical modification of siRNAs reduces off-target'' transcript silencing
Aimee L. Jackson et al.
RNA (2006)
Off-target effects by siRNA can induce toxic phenotype
Yuriy Fedorov et al.
RNA (2006)
RNAi-mediated gene silencing in non-human primates
TS Zimmermann et al.
NATURE (2006)
Targeted suppression of an amyloidogenic transthyretin with antisense oligonucleotides
MD Benson et al.
MUSCLE & NERVE (2006)
Insights into the kinetics of siRNA-mediated gene silencing from live-cell and live-animal bioluminescent imaging
DW Bartlett et al.
NUCLEIC ACIDS RESEARCH (2006)
Plasma protein binding of an antisense oligonucleotide targeting human ICAM-1 (ISIS 2302)
Tanya A. Watanabe et al.
OLIGONUCLEOTIDES (2006)
Early liver transplantation improves familial amyloidotic polyneuropathy patients' survival
OB Suhr et al.
AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS (2005)
Phosphorothioate-stimulated uptake of short interfering RNA by human cells
M Overhoff et al.
EMBO REPORTS (2005)
Activation of the mammalian immune system by siRNAs
JT Marques et al.
NATURE BIOTECHNOLOGY (2005)
Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs
DV Morrissey et al.
NATURE BIOTECHNOLOGY (2005)
Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors
EW Song et al.
NATURE BIOTECHNOLOGY (2005)
Sequence-specific potent induction of IFN-α by short interfering RNA in plasmacytoid dendritic cells through TLR7
V Hornung et al.
NATURE MEDICINE (2005)
Lactosylated poly(ethylene glycol)-siRNA conjugate through acid-labile ß-thiopropionate linkage to construct pH-sensitive polyion complex micelles achieving enhanced gene silencing in hepatoma cells
M Oishi et al.
JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2005)
Argonaute2 is the catalytic engine of mammalian RNAi
JD Liu et al.
SCIENCE (2004)
Crystal structure of argonaute and its implications for RISC slicer activity
JJ Song et al.
SCIENCE (2004)
Increased uptake of antisense oligonucleotides by delivery as double stranded complexes
A Astriab-Fisher et al.
BIOCHEMICAL PHARMACOLOGY (2004)
Human Argonaute2 mediates RNA cleavage targeted by miRNAs and siRNAs
G Meister et al.
MOLECULAR CELL (2004)
Selective binding to transthyretin and tetramer stabilization in serum from patients with familial amyloidotic polyneuropathy by an iodinated diflunisal derivative
MR Almeida et al.
BIOCHEMICAL JOURNAL (2004)
Functional siRNAs and rniRNAs exhibit strand bias
A Khvorova et al.
CELL (2003)
Activation of the interferon system by short-interfering RNAs
CA Sledz et al.
NATURE CELL BIOLOGY (2003)
siRNA function in RNAi: A chemical modification analysis
YL Chiu et al.
RNA (2003)
Molecular mechanisms of amyloidosis
G Merlini et al.
NEW ENGLAND JOURNAL OF MEDICINE (2003)
Expression profiling reveals off-target gene regulation by RNAi
AL Jackson et al.
NATURE BIOTECHNOLOGY (2003)
Gene silencing by systemic delivery of synthetic siRNAs in adult mice
DR Sorensen et al.
JOURNAL OF MOLECULAR BIOLOGY (2003)
Antisense technologies - Improvement through novel chemical modifications
J Kurreck
EUROPEAN JOURNAL OF BIOCHEMISTRY (2003)
RNA interference targeting Fas protects mice from fulminant hepatitis
EW Song et al.
NATURE MEDICINE (2003)
Tolerance for mutations and chemical modifications in a siRNA
M Amarzguioui et al.
NUCLEIC ACIDS RESEARCH (2003)
A microRNA in a multiple-turnover RNAi enzyme complex
G Hutvágner et al.
SCIENCE (2002)
Subcellular trafficking of antisense oligonucleotides and down-regulation of bcl-2 gene expression in human melanoma cells using a fusogenic liposome delivery system
Q Hu et al.
NUCLEIC ACIDS RESEARCH (2002)
Cellular uptake of antisense oligonucleotides after complexing or conjugation with cell-penetrating model peptides
J Oehlke et al.
EUROPEAN JOURNAL OF BIOCHEMISTRY (2002)
Control of alternative splicing by antisense oligonucleotides as a potential chemotherapy: effects on gene expression
DR Mercatante et al.
BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2002)
Design of antisense oligonucleotides stabilized by locked nucleic acids
J Kurreck et al.
NUCLEIC ACIDS RESEARCH (2002)
The high binding affinity of phosphorothioate-modified oligomers for Ff gene 5 protein is moderated by the addition of C-5 propyne or 2′-O-methyl modifications
TC Mou et al.
NUCLEIC ACIDS RESEARCH (2002)
A role for the RNase III enzyme DCR-1 in RNA interference and germ line development in Caenorhabditis elegans
SW Knight et al.
SCIENCE (2001)
Variant transthyretin in blood circulation can transverse the blood-cerebrospinal barrier: Qualitative analyses of transthyretin metabolism in sequential liver transplantation
H Terazaki et al.
TRANSPLANTATION (2001)
Transthyretin amyloidosis: a tale of weak interactions
MJM Saraiva
FEBS LETTERS (2001)
Role for a bidentate ribonuclease in the initiation step of RNA interference
E Bernstein et al.
NATURE (2001)
RNA interference is mediated by 21-and 22-nucleotide RNAs
SM Elbashir et al.
GENES & DEVELOPMENT (2001)
Inhibition of transthyretin-met30 expression using Inosine15.1-Hammerhead ribozymes in cell culture
MJ Pröpsting et al.
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2000)
The preaggregated state of an amyloidogenic protein: Hydrostatic pressure converts native transthyretin into the amyloidogenic state
AD Ferrao-Gonzales et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2000)
Nuclear gene targeting using negatively charged liposomes
C Welz et al.
INTERNATIONAL JOURNAL OF PHARMACEUTICS (2000)
Nucleocytoplasmic shuttling:: a novel in vivo property of antisense phosphorothioate oligodeoxynucleotides
P Lorenz et al.
NUCLEIC ACIDS RESEARCH (2000)
Share Paper
