4.6 Review

Antisense technology: A review

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Review Biochemistry & Molecular Biology

RNA toxicity in non-coding repeat expansion disorders

Bart Swinnen et al.

EMBO JOURNAL (2020)

Article Gastroenterology & Hepatology

Mongersen (GED-0301) for Active Crohn's Disease: Results of a Phase 3 Study

Bruce E. Sands et al.

AMERICAN JOURNAL OF GASTROENTEROLOGY (2020)

Review Pharmacology & Pharmacy

100 years of modelling ligand-receptor binding and response: A focus on GPCRs

David B. Finlay et al.

BRITISH JOURNAL OF PHARMACOLOGY (2020)

Article Medicine, General & Internal

Lipoprotein(a) Reduction in Persons with Cardiovascular Disease

Sotirios Tsimikas et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

Article Biochemistry & Molecular Biology

Golgi-endosome transport mediated by M6PR facilitates release of antisense oligonucleotides from endosomes

Xue-hai Liang et al.

NUCLEIC ACIDS RESEARCH (2020)

Article Biochemistry & Molecular Biology

Underlying Immune Disorder May Predispose Some Transthyretin Amyloidosis Subjects to Inotersen-Mediated Thrombocytopenia

PadmaKumar Narayanan et al.

NUCLEIC ACID THERAPEUTICS (2020)

Article Biochemistry & Molecular Biology

Phosphorothioate modified oligonucleotide-protein interactions

Stanley T. Crooke et al.

NUCLEIC ACIDS RESEARCH (2020)

Review Biotechnology & Applied Microbiology

Delivery of Oligonucleotides to the Liver with GalNAc: From Research to Registered Therapeutic Drug

Alexandre J. Debacker et al.

MOLECULAR THERAPY (2020)

Article Medicine, General & Internal

Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

Timothy Miller et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

Article Biochemistry & Molecular Biology

Gapmer Antisense Oligonucleotides Targeting 5S Ribosomal RNA Can Reduce Mature 5S Ribosomal RNA by Two Mechanisms

Adam J. Pollak et al.

NUCLEIC ACID THERAPEUTICS (2020)

Article Medicine, General & Internal

Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema

Danny M. Cohn et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

Review Medicine, Research & Experimental

Entering the Modern Era of Gene Therapy

Xavier M. Anguela et al.

ANNUAL REVIEW OF MEDICINE, VOL 70 (2019)

Review Medicine, Research & Experimental

Therapeutic Antisense Oligonucleotides Are Coming of Age

C. Frank Bennett

ANNUAL REVIEW OF MEDICINE, VOL 70 (2019)

Correction Cell Biology

RNA-Targeted Therapeutics (vol 27, pg 714, 2018)

Stanley T. Crooke et al.

CELL METABOLISM (2019)

Review Biotechnology & Applied Microbiology

The current state and future directions of RNAi-based therapeutics

Ryan L. Setten et al.

NATURE REVIEWS DRUG DISCOVERY (2019)

Article Biochemistry & Molecular Biology

Safety evaluation of 2-deoxy-2-fluoro nucleotides in GalNAc-siRNA conjugates

Maja M. Janas et al.

NUCLEIC ACIDS RESEARCH (2019)

Article Biochemistry & Molecular Biology

IONIS-PKKRx a Novel Antisense Inhibitor of Prekallikrein and Bradykinin Production

Jason D. Ferrone et al.

NUCLEIC ACID THERAPEUTICS (2019)

Article Cardiac & Cardiovascular Systems

N-acetyl galactosamine-conjugated antisense drug to APOC3 mRNA, triglycerides and atherogenic lipoprotein levels

Veronica J. Alexander et al.

EUROPEAN HEART JOURNAL (2019)

Article Biotechnology & Applied Microbiology

Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index

Wen Shen et al.

NATURE BIOTECHNOLOGY (2019)

Article Medicine, General & Internal

Targeting Huntingtin Expression in Patients with Huntington's Disease

Sarah J. Tabrizi et al.

NEW ENGLAND JOURNAL OF MEDICINE (2019)

Review Pharmacology & Pharmacy

Current Development of siRNA Bioconjugates: From Research to the Clinic

Ivan Chernikov et al.

FRONTIERS IN PHARMACOLOGY (2019)

Article Neurosciences

Antisense Oligonucleotide Therapies for Neurodegenerative Diseases

C. Frank Bennett et al.

Annual Review of Neuroscience (2019)

Article Medicine, General & Internal

Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy

Lindsay N. Alfano et al.

MEDICINE (2019)

Article Biochemistry & Molecular Biology

Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle

Thazha P. Prakash et al.

NUCLEIC ACIDS RESEARCH (2019)

Article Biochemistry & Molecular Biology

mRNA levels can be reduced by antisense oligonucleotides via no-go decay pathway

Xue-hai Liang et al.

NUCLEIC ACIDS RESEARCH (2019)

Article Biochemistry & Molecular Biology

Lipid Conjugates Enhance Endosomal Release of Antisense Oligonucleotides Into Cells

Shiyu Wang et al.

NUCLEIC ACID THERAPEUTICS (2019)

Meeting Abstract Cardiac & Cardiovascular Systems

Phase 1 Investigation of a Ligand-Conjugated Antisense Oligonucleotide with Increased Potency for the Treatment of Transthyretin Amyloidosis

Nicholas J. Viney et al.

JOURNAL OF CARDIAC FAILURE (2019)

Article Medicine, General & Internal

Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome

J. L. Witztum et al.

NEW ENGLAND JOURNAL OF MEDICINE (2019)

Article Biochemistry & Molecular Biology

Phosphorothioate Antisense Oligonucleotides Bind P-Body Proteins and Mediate P-Body Assembly

Ying Wang et al.

NUCLEIC ACID THERAPEUTICS (2019)

Article Biochemistry & Molecular Biology

Kinetic and subcellular analysis of PS-ASO/protein interactions with P54nrb and RNase H1

Timothy A. Vickers et al.

NUCLEIC ACIDS RESEARCH (2019)

Review Oncology

Therapeutic Prospects of mRNA-Based Gene Therapy for Glioblastoma

Xiangjun Tang et al.

FRONTIERS IN ONCOLOGY (2019)

Article Medicine, Research & Experimental

Next-Generation Sequencing Technologies

W. Richard McCombie et al.

COLD SPRING HARBOR PERSPECTIVES IN MEDICINE (2019)

Article Biochemistry & Molecular Biology

Translation can affect the antisense activity of RNase H1-dependent oligonucleotides targeting mRNAs

Xue-Hai Liang et al.

NUCLEIC ACIDS RESEARCH (2018)

Review Cell Biology

RNA-Targeted Therapeutics

Stanley T. Crooke et al.

CELL METABOLISM (2018)

Review Pharmacology & Pharmacy

Locked nucleic acid: modality, diversity, and drug discovery

Peter H. Hagedorn et al.

DRUG DISCOVERY TODAY (2018)

Article Endocrinology & Metabolism

A randomised, open-label, parallel group phase 2 study of antisense oligonucleotide therapy in acromegaly

Peter J. Trainer et al.

EUROPEAN JOURNAL OF ENDOCRINOLOGY (2018)

Review Infectious Diseases

Discovery and development of new antibacterial drugs: learning from experience?

Nicole Jackson et al.

JOURNAL OF ANTIMICROBIAL CHEMOTHERAPY (2018)

Article Medicine, General & Internal

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

M. D. Benson et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

Article Medicine, General & Internal

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

E. Mercuri et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

Article Biochemistry & Molecular Biology

The Effects of 2-O-Methoxyethyl Oligonucleotides on Renal Function in Humans

Stanley T. Crooke et al.

NUCLEIC ACID THERAPEUTICS (2018)

Article Medicine, Research & Experimental

Membrane Destabilization Induced by Lipid Species Increases Activity of Phosphorothioate-Antisense Oligonucleotides

Shiyu Wang et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2018)

Review Endocrinology & Metabolism

The Discovery of Insulin: An Important Milestone in the History of Medicine

Ignazio Vecchio et al.

FRONTIERS IN ENDOCRINOLOGY (2018)

Article Multidisciplinary Sciences

Targeted delivery of antisense oligonucleotides to pancreatic β-cells

C. Ammala et al.

SCIENCE ADVANCES (2018)

Editorial Material Pharmacology & Pharmacy

Alicaforsen for the treatment of inflammatory bowel disease

Vipul Jairath et al.

EXPERT OPINION ON INVESTIGATIONAL DRUGS (2017)

Article Biochemistry & Molecular Biology

A-44G transition in SMN2 intron 6 protects patients with spinal muscular atrophy

Xingxing Wu et al.

HUMAN MOLECULAR GENETICS (2017)

Article Immunology

Alicaforsen in the treatment of pouchitis

Thomas Greuter et al.

IMMUNOTHERAPY (2017)

Article Pharmacology & Pharmacy

RNA Therapeutics in Oncology: Advances, Challenges, and Future Directions

A. Robert MacLeod et al.

JOURNAL OF CLINICAL PHARMACOLOGY (2017)

Article Biotechnology & Applied Microbiology

Cellular uptake and trafficking of antisense oligonucleotides

Stanley T. Crooke et al.

NATURE BIOTECHNOLOGY (2017)

Article Medicine, General & Internal

Cardiovascular and Metabolic Effects of ANGPTL3 Antisense Oligonucleotides

Mark J. Graham et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

Article Medicine, General & Internal

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

R. S. Finkel et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

Article Biochemistry & Molecular Biology

The impact of base stacking on the conformations and electrostatics of single-stranded DNA

Alex Plumridge et al.

NUCLEIC ACIDS RESEARCH (2017)

Article Biochemistry & Molecular Biology

Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc-siRNA conjugates

Jayaprakash K. Nair et al.

NUCLEIC ACIDS RESEARCH (2017)

Article Biochemistry & Molecular Biology

Nucleic acid binding proteins affect the subcellular distribution of phosphorothioate antisense oligonucleotides

Jeffrey K. Bailey et al.

NUCLEIC ACIDS RESEARCH (2017)

Article Biochemistry & Molecular Biology

The Effects of 2′-O-Methoxyethyl Containing Antisense Oligonucleotides on Platelets in Human Clinical Trials

Stanley T. Crooke et al.

NUCLEIC ACID THERAPEUTICS (2017)

Article Biochemistry & Molecular Biology

Molecular Mechanisms of Antisense Oligonucleotides

Stanley T. Crooke

NUCLEIC ACID THERAPEUTICS (2017)

Article Multidisciplinary Sciences

Transcriptomic analysis of the role of RasGEF1B circular RNA in the TLR4/LPS pathway

Wei Lun Ng et al.

SCIENTIFIC REPORTS (2017)

Review Biochemistry & Molecular Biology

Antisense inhibition of apolipoprotein (a) to lower plasma lipoprotein (a) levels in humans

Mark J. Graham et al.

JOURNAL OF LIPID RESEARCH (2016)

Article Biotechnology & Applied Microbiology

Translation efficiency of mRNAs is increased by antisense oligonucleotides targeting upstream open reading frames

Xue-hai Liang et al.

NATURE BIOTECHNOLOGY (2016)

Article Biochemistry & Molecular Biology

Viable RNaseH1 knockout mice show RNaseH1 is essential for R loop processing, mitochondrial and liver function

Walt F. Lima et al.

NUCLEIC ACIDS RESEARCH (2016)

Article Multidisciplinary Sciences

Development of a Quantitative BRET Affinity Assay for Nucleic Acid-Protein Interactions

Timothy A. Vickers et al.

PLOS ONE (2016)

Article Pharmacology & Pharmacy

Renal Effects of Antisense-Mediated Inhibition of SGLT2

Leonie van Meer et al.

JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS (2016)

Review Pharmacology & Pharmacy

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

Richard S. Geary et al.

ADVANCED DRUG DELIVERY REVIEWS (2015)

Article Clinical Neurology

Spinal Muscular Atrophy

Stephen J. Kolb et al.

NEUROLOGIC CLINICS (2015)

Article Medicine, General & Internal

Factor XI Antisense Oligonucleotide for Prevention of Venous Thrombosis

Harry R. Bueller et al.

NEW ENGLAND JOURNAL OF MEDICINE (2015)

Article Biochemistry & Molecular Biology

A cytoplasmic pathway for gapmer antisense oligonucleotide-mediated gene silencing in mammalian cells

Daniela Castanotto et al.

NUCLEIC ACIDS RESEARCH (2015)

Article Biochemistry & Molecular Biology

Identification and characterization of intracellular proteins that bind oligonucleotides with phosphorothioate linkages

Xue-hai Liang et al.

NUCLEIC ACIDS RESEARCH (2015)

Article Biochemistry & Molecular Biology

2′-Fluoro-modified phosphorothioate oligonucleotide can cause rapid degradation of P54nrb and PSF

Wen Shen et al.

NUCLEIC ACIDS RESEARCH (2015)

Article Multidisciplinary Sciences

Stereochemical bias introduced during RNA synthesis modulates the activity of phosphorothioate siRNAs

Hartmut Jahns et al.

NATURE COMMUNICATIONS (2015)

Article Multidisciplinary Sciences

Substitution of tryptophan 89 with tyrosine switches the DNA binding mode of PC4

Jinguang Huang et al.

SCIENTIFIC REPORTS (2015)

Article Pharmacology & Pharmacy

Long-term safety and efficacy of microRNA-targeted therapy in chronic hepatitis C patients

Meike H. van der Ree et al.

ANTIVIRAL RESEARCH (2014)

Article Biochemistry & Molecular Biology

Phosphorothioate oligonucleotides can displace NEAT1 RNA and form nuclear paraspeckle-like structures

Wen Shen et al.

NUCLEIC ACIDS RESEARCH (2014)

Article Biochemistry & Molecular Biology

Transfection of siRNAs can alter miRNA levels and trigger non-specific protein degradation in mammalian cells

Xue-hai Liang et al.

BIOCHIMICA ET BIOPHYSICA ACTA-GENE REGULATORY MECHANISMS (2013)

Review Biotechnology & Applied Microbiology

Advances in the development of nucleoside and nucleotide analogues for cancer and viral diseases

Lars Petter Jordheim et al.

NATURE REVIEWS DRUG DISCOVERY (2013)

Article Medicine, General & Internal

Treatment of HCV Infection by Targeting MicroRNA

Harry L. A. Janssen et al.

NEW ENGLAND JOURNAL OF MEDICINE (2013)

Article Biochemistry & Molecular Biology

Design, synthesis and biological evaluation of novel antagonist compounds of Toll-like receptors 7, 8 and 9

Ekambar R. Kandimalla et al.

NUCLEIC ACIDS RESEARCH (2013)

Review Genetics & Heredity

Gene Expression Regulation by Upstream Open Reading Frames and Human Disease

Cristina Barbosa et al.

PLOS GENETICS (2013)

Article Biochemistry & Molecular Biology

Single-Stranded siRNAs Activate RNAi in Animals

Walt F. Lima et al.

Review Biochemistry & Molecular Biology

The Nicotinic Acetylcholine Receptor: The Founding Father of the Pentameric Ligand-gated Ion Channel Superfamily

Jean-Pierre Changeux

JOURNAL OF BIOLOGICAL CHEMISTRY (2012)

Article Biochemistry & Molecular Biology

Aptamer-Mediated Delivery of Splice-Switching Oligonucleotides to the Nuclei of Cancer Cells

Jonathan W. Kotula et al.

NUCLEIC ACID THERAPEUTICS (2012)

Article Chemistry, Multidisciplinary

Unique Gene-Silencing and Structural Properties of 2 '-Fluoro-Modified siRNAs

Muthiah Manoharan et al.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2011)

Article Biochemistry & Molecular Biology

Efficient and specific knockdown of small non-coding RNAs in mammalian cells and in mice

Xue-hai Liang et al.

NUCLEIC ACIDS RESEARCH (2011)

Article Biochemistry & Molecular Biology

U1 adaptors result in reduction of multiple pre-mRNA species principally by sequestering U1snRNP

Timothy A. Vickers et al.

NUCLEIC ACIDS RESEARCH (2011)

Article Biochemistry & Molecular Biology

An Upstream Open Reading Frame and the Context of the Two AUG Codons Affect the Abundance of Mitochondrial and Nuclear RNase H1

Yutaka Suzuki et al.

MOLECULAR AND CELLULAR BIOLOGY (2010)

Review Biotechnology & Applied Microbiology

Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application

Aimee L. Jackson et al.

NATURE REVIEWS DRUG DISCOVERY (2010)

Article Chemistry, Medicinal

Discovery of Innovative Small Molecule Therapeutics

Magid Abou-Gharbia

JOURNAL OF MEDICINAL CHEMISTRY (2009)

Article Genetics & Heredity

Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in Transgenic mice

Yimin Hua et al.

AMERICAN JOURNAL OF HUMAN GENETICS (2008)

Article Chemistry, Medicinal

Oral delivery of antisense oligonucleotides in man

Lloyd G. Tillman et al.

JOURNAL OF PHARMACEUTICAL SCIENCES (2008)

Article Biochemistry & Molecular Biology

Enhancement of SMN2 Exon 7 inclusion by antisense oligonucleotides targeting the exon

Yimin Hua et al.

PLOS BIOLOGY (2007)

Article Biochemistry & Molecular Biology

Anti-inflammatory activity of inhaled IL-4 receptor-α antisense oligonucleotide in mice

James G. Karras et al.

AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY (2007)

Article Pharmacology & Pharmacy

Human RNase H1 discriminates between subtle variations in the structure of the heteroduplex substrate

Walt F. Lima et al.

MOLECULAR PHARMACOLOGY (2007)

Article Biochemistry & Molecular Biology

Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals

Eric E. Swayze et al.

NUCLEIC ACIDS RESEARCH (2007)

Article Biochemistry & Molecular Biology

A global transcription cofactor bound to juxtaposed strands of unwound DNA

S Werten et al.

NATURE STRUCTURAL & MOLECULAR BIOLOGY (2006)

Article Biochemistry & Molecular Biology

Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs

HJ Wu et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2004)

Article Biochemistry & Molecular Biology

Structural requirements at the catalytic site of the heteroduplex substrate for human RNase H1 catalysis

WF Lima et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2004)

Article Chemistry, Multidisciplinary

An oxazaphospholidine approach for the stereocontrolled synthesis of oligonucleoside phosphorothioates

N Oka et al.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2003)

Article Biochemistry & Molecular Biology

Efficient reduction of target RNAs by small interfering RNA and RNase H-dependent antisense agents - A comparative analysis

TA Vickers et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2003)

Article Pharmacology & Pharmacy

Phase I trial of ISIS 104838, a 2′-methoxyethyl modified antisense oligonucleotide targeting tumor necrosis factor-α

KL Sewell et al.

JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS (2002)

Article Biotechnology & Applied Microbiology

The emergence of the drug receptor theory

AH Maehle et al.

NATURE REVIEWS DRUG DISCOVERY (2002)

Article Biochemistry & Molecular Biology

Paraspeckles: A novel nuclear domain

AH Fox et al.

CURRENT BIOLOGY (2002)

Article Genetics & Heredity

Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2

MD Mailman et al.

GENETICS IN MEDICINE (2002)

Article Biochemistry & Molecular Biology

Investigating the structure of human RNase H1 by site-directed mutagenesis

HJ Wu et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2001)

Review Pharmacology & Pharmacy

Fomivirsen

LR Grillone et al.

DRUGS OF TODAY (2001)

Article Biochemistry & Molecular Biology

Fully modified 2′ MOE oligonucleotides redirect polyadenylation

TA Vickers et al.

NUCLEIC ACIDS RESEARCH (2001)

Article Biochemistry & Molecular Biology

Variations in mRNA content have no effect on the potency of antisense oligonucleotides

L Miraglia et al.

ANTISENSE & NUCLEIC ACID DRUG DEVELOPMENT (2000)

Review Biochemistry & Molecular Biology

Phosphorothioate oligodeoxynucleotides: What is their origin and what is unique about them?

F Eckstein

ANTISENSE & NUCLEIC ACID DRUG DEVELOPMENT (2000)