The powerful world of antisense oligonucleotides: From bench to bedside

Lipoprotein(a) Reduction in Persons with Cardiovascular Disease

(2020) Sotirios Tsimikas et al.   NEW ENGLAND JOURNAL OF MEDICINE

Bioconjugated oligonucleotides: recent developments and therapeutic applications

(2019) sebastien benizri et al.   BIOCONJUGATE CHEMISTRY

Circular RNA circNRIP1 acts as a microRNA-149-5p sponge to promote gastric cancer progression via the AKT1/mTOR pathway

(2019) Xing Zhang et al.   Molecular Cancer

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

(2019) Wen Shen et al.   NATURE BIOTECHNOLOGY

The Clinical Potential of Oligonucleotide Therapeutics against Pancreatic Cancer

(2019) Kazuki Takakura et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Pivotal Trial of Enfortumab Vedotin in Urothelial Carcinoma After Platinum and Anti-Programmed Death 1/Programmed Death Ligand 1 Therapy

(2019) Jonathan E. Rosenberg et al.   JOURNAL OF CLINICAL ONCOLOGY

Antisense oligonucleotide eluforsen is safe and improves respiratory symptoms in F508DEL cystic fibrosis

(2019) Pavel Drevinek et al.   Journal of Cystic Fibrosis

Noncoding RNAs in cancer therapy resistance and targeted drug development

(2019) Wen-Tao Wang et al.   Journal of Hematology & Oncology

Enhanced Potency of GalNAc-Conjugated Antisense Oligonucleotides in Hepatocellular Cancer Models

(2019) Youngsoo Kim et al.   MOLECULAR THERAPY

Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle

(2019) Thazha P Prakash et al.   NUCLEIC ACIDS RESEARCH

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

(2019) Xue-hai Liang et al.   NUCLEIC ACIDS RESEARCH

A gapmer antisense oligonucleotide targeting SRRM4 is a novel therapeutic medicine for lung cancer

(2019) Masahito Shimojo et al.   Scientific Reports

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

(2019) Jinkuk Kim et al.   NEW ENGLAND JOURNAL OF MEDICINE

Locked nucleic acid: modality, diversity, and drug discovery

(2018) Peter H. Hagedorn et al.   DRUG DISCOVERY TODAY

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

(2018) Merrill D. Benson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

(2018) Eugenio Mercuri et al.   NEW ENGLAND JOURNAL OF MEDICINE

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

(2018) Alexis A. Thompson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy

(2018) Alison M. Blain et al.   PLoS One

Anillin is required for tumor growth and regulated by miR-15a/miR-16-1 in HBV-related hepatocellular carcinoma

(2018) Yi-Fan Lian et al.   Aging-US

Inotersen: First Global Approval

(2018) Susan J. Keam   DRUGS

Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis

(2018) Isabelle Sermet-Gaudelus et al.   Journal of Cystic Fibrosis

NTRK fusion-positive cancers and TRK inhibitor therapy

(2018) Emiliano Cocco et al.   Nature Reviews Clinical Oncology

Drugging the RNA World

(2018) Matthew D. Disney et al.   Cold Spring Harbor Perspectives in Biology

Integrated Assessment of the Clinical Performance of GalNAc3-Conjugated 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides: I. Human Volunteer Experience

(2018) Stanley T. Crooke et al.   Nucleic Acid Therapeutics

STAT3 antisense oligonucleotide AZD9150 in a subset of patients with heavily pretreated lymphoma: results of a phase 1b trial

(2018) Matthew J. Reilley et al.   Journal for ImmunoTherapy of Cancer

Primary tumor site and anti-EGFR monoclonal antibody benefit in metastatic colorectal cancer: a meta-analysis

(2017) Dandan Li et al.   Future Oncology

Custirsen in combination with docetaxel and prednisone for patients with metastatic castration-resistant prostate cancer (SYNERGY trial): a phase 3, multicentre, open-label, randomised trial

(2017) Kim N Chi et al.   LANCET ONCOLOGY

Definition and identification of small RNA sponges: Focus on miRNA sequestration

(2017) Mélodie Migault et al.   METHODS

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

A non-coding function of TYRP1 mRNA promotes melanoma growth

(2017) David Gilot et al.   NATURE CELL BIOLOGY

MicroRNA therapeutics: towards a new era for the management of cancer and other diseases

(2017) Rajesha Rupaimoole et al.   NATURE REVIEWS DRUG DISCOVERY

Antisense oligonucleotides: the next frontier for treatment of neurological disorders

(2017) Carlo Rinaldi et al.   Nature Reviews Neurology

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9

(2017) Kevin Fitzgerald et al.   NEW ENGLAND JOURNAL OF MEDICINE

Inclisiran in Patients at High Cardiovascular Risk with Elevated LDL Cholesterol

(2017) Kausik K. Ray et al.   NEW ENGLAND JOURNAL OF MEDICINE

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes

(2017) Michael Tanowitz et al.   NUCLEIC ACIDS RESEARCH

Antisense oligonucleotides targeting translation inhibitory elements in 5′ UTRs can selectively increase protein levels

(2017) Xue-hai Liang et al.   NUCLEIC ACIDS RESEARCH

Targeted therapy in severe asthma today: focus on immunoglobulin E

(2017) Girolamo Pelaia et al.   Drug Design Development and Therapy

Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics

(2017) Yuanyu Huang   Molecular Therapy-Nucleic Acids

Therapeutic miRNA and siRNA: Moving from Bench to Clinic as Next Generation Medicine

(2017) Chiranjib Chakraborty et al.   Molecular Therapy-Nucleic Acids

Lentiviral vectors can be used for full-length dystrophin gene therapy

(2017) John R. Counsell et al.   Scientific Reports

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

(2016) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Inhibition of STAT3 with the Generation 2.5 Antisense Oligonucleotide, AZD9150, Decreases Neuroblastoma Tumorigenicity and Increases Chemosensitivity

(2016) Seiichi Odate et al.   CLINICAL CANCER RESEARCH

Antisense molecules: A new class of drugs

(2016) Daniel P. Potaczek et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Melanoma addiction to the long non-coding RNA SAMMSON

(2016) Eleonora Leucci et al.   NATURE

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

(2016) Xue-hai Liang et al.   NATURE BIOTECHNOLOGY

A microRNA switch regulates the rise in hypothalamic GnRH production before puberty

(2016) Andrea Messina et al.   NATURE NEUROSCIENCE

Efficient Suppression of Hepatitis C Virus Replication by Combination Treatment with miR-122 Antagonism and Direct-acting Antivirals in Cell Culture Systems

(2016) Fanwei Liu et al.   Scientific Reports

Miravirsen dosing in chronic hepatitis C patients results in decreased microRNA-122 levels without affecting other microRNAs in plasma

(2015) M. H. van der Ree et al.   ALIMENTARY PHARMACOLOGY & THERAPEUTICS

Mipomersen, an Antisense Oligonucleotide to Apolipoprotein B-100, Reduces Lipoprotein(a) in Various Populations With HypercholesterolemiaSignificance

(2015) Raul D. Santos et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Differentiation of mammary tumors and reduction in metastasis uponMalat1lncRNA loss

(2015) Gayatri Arun et al.   GENES & DEVELOPMENT

Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

(2015) Aurélie Goyenvalle et al.   NATURE MEDICINE

The emerging role of lncRNAs in cancer

(2015) Maite Huarte   NATURE MEDICINE

AZD9150, a next-generation antisense oligonucleotide inhibitor of STAT3 with early evidence of clinical activity in lymphoma and lung cancer

(2015) David Hong et al.   Science Translational Medicine

Duchenne muscular dystrophy: current cell therapies

(2015) Dorota Sienkiewicz et al.   Therapeutic Advances in Neurological Disorders

Towards a therapy for Angelman syndrome by targeting a long non-coding RNA

(2014) Linyan Meng et al.   NATURE

A day in the life of the spliceosome

(2014) A. Gregory Matera et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

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

DNA-based digital tension probes reveal integrin forces during early cell adhesion

(2014) Yun Zhang et al.   Nature Communications

Mipomersen Sodium: First Global Approval

(2013) Philip Hair et al.   DRUGS

Gene therapy on the move

(2013) Kerstin B. Kaufmann et al.   EMBO Molecular Medicine

MicroRNAs and other non-coding RNAs as targets for anticancer drug development

(2013) Hui Ling et al.   NATURE REVIEWS DRUG DISCOVERY

T.I.2 Exon skipping and PRO044 in Duchenne muscular dystrophy: Extending the program

(2013) A. Ferlini et al.   NEUROMUSCULAR DISORDERS

Treatment of HCV Infection by Targeting MicroRNA

(2013) Harry L.A. Janssen et al.   NEW ENGLAND JOURNAL OF MEDICINE

Single-molecule study of the CUG repeat–MBNL1 interaction and its inhibition by small molecules

(2013) Amin Haghighat Jahromi et al.   NUCLEIC ACIDS RESEARCH

The Noncoding RNA MALAT1 Is a Critical Regulator of the Metastasis Phenotype of Lung Cancer Cells

(2012) T. Gutschner et al.   CANCER RESEARCH

Mipomersen

(2012) Daniel N. Ricotta et al.   Cardiology in Review

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

(2012) Thurman M. Wheeler et al.   NATURE

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

(2012) Holly B. Kordasiewicz et al.   NEURON

Mechanisms of activation of the paternally expressed genes by the Prader-Willi imprinting center in the Prader-Willi/Angelman syndromes domains

(2012) S. Rabinovitz et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment

(2012) Corinne Betts et al.   Molecular Therapy-Nucleic Acids

Efficacy of apolipoprotein B synthesis inhibition in subjects with mild-to-moderate hyperlipidaemia

(2011) Fatima Akdim et al.   EUROPEAN HEART JOURNAL

Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model

(2011) Yimin Hua et al.   NATURE

Targeted therapies: how personal should we go?

(2011) Miriam Martini et al.   Nature Reviews Clinical Oncology

Improved Survival with Vemurafenib in Melanoma with BRAF V600E Mutation

(2011) Paul B. Chapman et al.   NEW ENGLAND JOURNAL OF MEDICINE

Angelman syndrome: insights into genomic imprinting and neurodevelopmental phenotypes

(2011) Angela M. Mabb et al.   TRENDS IN NEUROSCIENCES

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

(2010) Y. Hua et al.   GENES & DEVELOPMENT

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

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

Progression of transthyretin amyloid neuropathy after liver transplantation

(2010) J. J. Liepnieks et al.   NEUROLOGY

A Locked Nucleic Acid Antisense Oligonucleotide (LNA) Silences PCSK9 and Enhances LDLR Expression In Vitro and In Vivo

(2010) Nidhi Gupta et al.   PLoS One

Locked nucleic acid as a novel class of therapeutic agents

(2010) Rakesh N. Veedu et al.   RNA Biology

No-go decay: a quality control mechanism for RNA in translation

(2010) Yuriko Harigaya et al.   Wiley Interdisciplinary Reviews-RNA

Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers†

(2009) Natee Jearawiriyapaisarn et al.   CARDIOVASCULAR RESEARCH

MicroRNAs: Target Recognition and Regulatory Functions

(2009) David P. Bartel   CELL

Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort

(2009) Kevin M. Flanigan et al.   HUMAN MUTATION

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

The HER-2 Receptor and Breast Cancer: Ten Years of Targeted Anti-HER-2 Therapy and Personalized Medicine

(2009) J. S. Ross et al.   ONCOLOGIST

Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy

(2009) S. A. M. Mulders et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Therapeutic Silencing of MicroRNA-122 in Primates with Chronic Hepatitis C Virus Infection

(2009) R. E. Lanford et al.   SCIENCE

Expanded CTG repeats within the DMPK 3' UTR causes severe skeletal muscle wasting in an inducible mouse model for myotonic dystrophy

(2008) J. P. Orengo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA