Non-coding RNAs as drug targets

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

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

Approaches to Validate and Manipulate RNA Targets with Small Molecules in Cells

(2016) Jessica L. Childs-Disney et al.   Annual Review of Pharmacology and Toxicology

MALAT1 long non-coding RNA in cancer

(2016) Rei Yoshimoto et al.   Biochimica et Biophysica Acta-Gene Regulatory Mechanisms

Circular RNAs: Identification, biogenesis and function

(2016) Karoline K. Ebbesen et al.   Biochimica et Biophysica Acta-Gene Regulatory Mechanisms

Noncoding RNA NORAD Regulates Genomic Stability by Sequestering PUMILIO Proteins

(2016) Sungyul Lee et al.   CELL

Disease-associated repeat instability and mismatch repair

(2016) Monika H.M. Schmidt et al.   DNA REPAIR

Intrathecal Injections in Children With Spinal Muscular Atrophy

(2016) Manon Haché et al.   JOURNAL OF CHILD NEUROLOGY

Melanoma addiction to the long non-coding RNA SAMMSON

(2016) Eleonora Leucci et al.   NATURE

Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy

(2016) Claudia A. Chiriboga et al.   NEUROLOGY

Functional annotation of the vlinc class of non-coding RNAs using systems biology approach

(2016) Georges St. Laurent et al.   NUCLEIC ACIDS RESEARCH

Activating frataxin expression by repeat-targeted nucleic acids

(2016) Liande Li et al.   Nature Communications

Cellular uptake and intracellular trafficking of oligonucleotides

(2015) R.L. Juliano et al.   ADVANCED DRUG DELIVERY REVIEWS

An overview of microRNAs

(2015) Scott M. Hammond   ADVANCED DRUG DELIVERY REVIEWS

Preclinical and clinical development of siRNA-based therapeutics

(2015) Gulnihal Ozcan et al.   ADVANCED DRUG DELIVERY REVIEWS

Exon skipping therapy for Duchenne muscular dystrophy

(2015) Ryszard Kole et al.   ADVANCED DRUG DELIVERY REVIEWS

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

miR-122 is a Unique Molecule with Great Potential in Diagnosis, Prognosis of Liver Disease, and Therapy Both as miRNA Mimic and Antimir

(2015) Sharda Thakral et al.   CURRENT GENE THERAPY

An overview of the clinical application of antisense oligonucleotides for RNA-targeting therapies

(2015) Graham McClorey et al.   CURRENT OPINION IN PHARMACOLOGY

Oligonucleotide therapeutics: chemistry, delivery and clinical progress

(2015) Vivek K Sharma et al.   Future Medicinal Chemistry

Differentiation of mammary tumors and reduction in metastasis uponMalat1lncRNA loss

(2015) Gayatri Arun et al.   GENES & DEVELOPMENT

Abstract C43: Safety, tolerability, and clinical activity of MRX34, the first-in-class liposomal miR-34 mimic, in patients with advanced solid tumors

(2015) Muhammad Shaalan Beg et al.   MOLECULAR CANCER THERAPEUTICS

Toward a Consensus on the Binding Specificity and Promiscuity of PRC2 for RNA

(2015) Chen Davidovich et al.   MOLECULAR CELL

Towards a molecular understanding of microRNA-mediated gene silencing

(2015) Stefanie Jonas et al.   NATURE REVIEWS GENETICS

Spinal muscular atrophy—recent therapeutic advances for an old challenge

(2015) Irene Faravelli et al.   Nature Reviews Neurology

Cellular localization of long non-coding RNAs affects silencing by RNAi more than by antisense oligonucleotides

(2015) Kim A. Lennox et al.   NUCLEIC ACIDS RESEARCH

Regulation of mammalian transcription and splicing by Nuclear RNAi

(2015) Roya Kalantari et al.   NUCLEIC ACIDS RESEARCH

Nucleic acid-binding specificity of human FUS protein

(2015) Xueyin Wang et al.   NUCLEIC ACIDS RESEARCH

MicroRNA-based therapy and breast cancer: A comprehensive review of novel therapeutic strategies from diagnosis to treatment

(2015) Parham Jabbarzadeh Kaboli et al.   PHARMACOLOGICAL RESEARCH

The recruitment of chromatin modifiers by long noncoding RNAs: lessons from PRC2

(2015) Chen Davidovich et al.   RNA

The Landscape of long noncoding RNA classification

(2015) Georges St. Laurent et al.   TRENDS IN GENETICS

Digital Quantitation of Potential Therapeutic Target RNAs

(2015) David W. Dodd et al.   Nucleic Acid Therapeutics

miR-34: from bench to bedside

(2015) Massimiliano Agostini et al.   Oncotarget

In VitroAntiviral Activity and Preclinical and Clinical Resistance Profile of Miravirsen, a Novel Anti-Hepatitis C Virus Therapeutic Targeting the Human Factor miR-122

(2014) Søren Ottosen et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

The Increasing Urgency for Standards in Basic Biologic Research

(2014) L. P. Freedman et al.   CANCER RESEARCH

RNA-RNA Interactions Enable Specific Targeting of Noncoding RNAs to Nascent Pre-mRNAs and Chromatin Sites

(2014) Jesse M. Engreitz et al.   CELL

Oral Druggable Space beyond the Rule of 5: Insights from Drugs and Clinical Candidates

(2014) Bradley Croy Doak et al.   CHEMISTRY & BIOLOGY

Lomitapide and Mipomersen

(2014) Daniel J. Rader et al.   CIRCULATION

TGF- -Induced Upregulation of malat1 Promotes Bladder Cancer Metastasis by Associating with suz12

(2014) Y. Fan et al.   CLINICAL CANCER RESEARCH

Personalized gene silencing therapeutics for Huntington disease

(2014) C. Kay et al.   CLINICAL GENETICS

Development of microRNA therapeutics is coming of age

(2014) E. van Rooij et al.   EMBO Molecular Medicine

Anti–microRNA-21 oligonucleotides prevent Alport nephropathy progression by stimulating metabolic pathways

(2014) Ivan G. Gomez et al.   JOURNAL OF CLINICAL INVESTIGATION

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

(2014) Thomas Voit et al.   LANCET NEUROLOGY

Dacarbazine with or without oblimersen (a Bcl-2 antisense oligonucleotide) in chemotherapy-naive patients with advanced melanoma and low–normal serum lactate dehydrogenase

(2014) Agop Y. Bedikian et al.   MELANOMA RESEARCH

The Long Noncoding RNAs NEAT1 and MALAT1 Bind Active Chromatin Sites

(2014) Jason A. West et al.   MOLECULAR CELL

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

(2014) Linyan Meng et al.   NATURE

Analysis of nuclear RNA interference in human cells by subcellular fractionation and Argonaute loading

(2014) Keith T Gagnon et al.   Nature Protocols

Therapeutic targeting of microRNAs: current status and future challenges

(2014) Zhonghan Li et al.   NATURE REVIEWS DRUG DISCOVERY

The rise of regulatory RNA

(2014) Kevin V. Morris et al.   NATURE REVIEWS GENETICS

Oligonucleotide-based strategies to combat polyglutamine diseases

(2014) A. Fiszer et al.   NUCLEIC ACIDS RESEARCH

Drug target miRNAs: chances and challenges

(2014) Marco F. Schmidt   TRENDS IN BIOTECHNOLOGY

RNAi Factors Are Present and Active in Human Cell Nuclei

(2014) Keith T. Gagnon et al.   Cell Reports

R-loops Associated with Triplet Repeat Expansions Promote Gene Silencing in Friedreich Ataxia and Fragile X Syndrome

(2014) Matthias Groh et al.   PLoS Genetics

What Can We Learn From Clinical Trials of Exon Skipping for DMD?

(2014) Qi-long Lu et al.   Molecular Therapy-Nucleic Acids

The MicroRNA Biology of the Mammalian Nucleus

(2014) Thomas C Roberts   Molecular Therapy-Nucleic Acids

Epigenetic-based therapies for Friedreich ataxia

(2014) Chiranjeevi Sandi et al.   Frontiers in Genetics

Molecular Mechanisms of RNA Interference

(2013) Ross C. Wilson et al.   Annual Review of Biophysics

Long non-coding RNAs: modulators of nuclear structure and function

(2013) Jan H Bergmann et al.   CURRENT OPINION IN CELL BIOLOGY

Guided by RNAs: X-Inactivation as a Model for lncRNA Function

(2013) John E. Froberg et al.   JOURNAL OF MOLECULAR BIOLOGY

Occupying Chromatin: Polycomb Mechanisms for Getting to Genomic Targets, Stopping Transcriptional Traffic, and Staying Put

(2013) Jeffrey A. Simon et al.   MOLECULAR CELL

Frontiers and Approaches to Chemical Synthesis of Oligodeoxyribonucleotides

(2013) Tatyana Abramova   MOLECULES

Rescue of hearing and vestibular function by antisense oligonucleotides in a mouse model of human deafness

(2013) Jennifer J Lentz et al.   NATURE MEDICINE

Targeting long non-coding RNA to therapeutically upregulate gene expression

(2013) Claes Wahlestedt   NATURE REVIEWS DRUG DISCOVERY

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

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

Promiscuous RNA binding by Polycomb repressive complex 2

(2013) Chen Davidovich et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

The Unstable Repeats—Three Evolving Faces of Neurological Disease

(2013) David L. Nelson et al.   NEURON

Treatment of HCV Infection by Targeting MicroRNA

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

Promoter RNA links transcriptional regulation of inflammatory pathway genes

(2013) Masayuki Matsui et al.   NUCLEIC ACIDS RESEARCH

Miravirsen (SPC3649) can inhibit the biogenesis of miR-122

(2013) Luca F. R. Gebert et al.   NUCLEIC ACIDS RESEARCH

A Chemical View of Oligonucleotides for Exon Skipping and Related Drug Applications

(2013) Peter Järver et al.   Nucleic Acid Therapeutics

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

(2012) T. Gutschner et al.   CANCER RESEARCH

Designing Chemically Modified Oligonucleotides for Targeted Gene Silencing

(2012) Glen F. Deleavey et al.   CHEMISTRY & BIOLOGY

Ube3a-ATS is an atypical RNA polymerase II transcript that represses the paternal expression of Ube3a

(2012) Linyan Meng et al.   HUMAN MOLECULAR GENETICS

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

(2012) Thurman M. Wheeler et al.   NATURE

Landscape of transcription in human cells

(2012) Sarah Djebali et al.   NATURE

Raise standards for preclinical cancer research

(2012) C. Glenn Begley et al.   NATURE

Inhibition of natural antisense transcripts in vivo results in gene-specific transcriptional upregulation

(2012) Farzaneh Modarresi et al.   NATURE BIOTECHNOLOGY

Synthetic oligonucleotides recruit ILF2/3 to RNA transcripts to modulate splicing

(2012) Frank Rigo et al.   Nature Chemical Biology

Loss of the abundant nuclear non-coding RNAMALAT1is compatible with life and development

(2012) Moritz Eißmann et al.   RNA Biology

Malat1 is not an essential component of nuclear speckles in mice

(2012) S. Nakagawa et al.   RNA

The lncRNA Malat1 Is Dispensable for Mouse Development but Its Transcription Plays a cis-Regulatory Role in the Adult

(2012) Bin Zhang et al.   Cell Reports

Oligonucleotide therapeutic approaches for Huntington disease

(2011) Dinah W.Y. Sah et al.   JOURNAL OF CLINICAL INVESTIGATION

Silencing disease genes in the laboratory and the clinic

(2011) Jonathan K Watts et al.   JOURNAL OF PATHOLOGY

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

Expanding the action of duplex RNAs into the nucleus: redirecting alternative splicing

(2011) Jing Liu et al.   NUCLEIC ACIDS RESEARCH

Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

(2011) M. A. Passini et al.   Science Translational Medicine

Response to “The Reality of Pervasive Transcription”

(2011) Harm van Bakel et al.   PLOS BIOLOGY

The Reality of Pervasive Transcription

(2011) Michael B. Clark et al.   PLOS BIOLOGY

RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

(2010) C. Frank Bennett et al.   Annual Review of Pharmacology and Toxicology

Off-Target Effects Related to the Phosphorothioate Modification of Nucleic Acids

(2010) Johannes Winkler et al.   ChemMedChem

Frequency of Usher syndrome in two pediatric populations: Implications for genetic screening of deaf and hard of hearing children

(2010) William J Kimberling et al.   GENETICS IN MEDICINE

Mitochondrial p32 Protein Is a Critical Regulator of Tumor Metabolism via Maintenance of Oxidative Phosphorylation

(2010) V. Fogal et al.   MOLECULAR AND CELLULAR BIOLOGY

The Nuclear-Retained Noncoding RNA MALAT1 Regulates Alternative Splicing by Modulating SR Splicing Factor Phosphorylation

(2010) Vidisha Tripathi et al.   MOLECULAR CELL

Repeat expansion disease: progress and puzzles in disease pathogenesis

(2010) Albert R. La Spada et al.   NATURE REVIEWS GENETICS

Locked nucleic acids (LNAs) reveal sequence requirements and kinetics of Xist RNA localization to the X chromosome

(2010) K. Sarma et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Most “Dark Matter” Transcripts Are Associated With Known Genes

(2010) Harm van Bakel et al.   PLOS BIOLOGY

Why the need for qPCR publication guidelines?—The case for MIQE

(2009) Stephen A. Bustin   METHODS

Chromatin signature reveals over a thousand highly conserved large non-coding RNAs in mammals

(2009) Mitchell Guttman et al.   NATURE

Efficient gene silencing by delivery of locked nucleic acid antisense oligonucleotides, unassisted by transfection reagents

(2009) C. A. Stein et al.   NUCLEIC ACIDS RESEARCH

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

Many human large intergenic noncoding RNAs associate with chromatin-modifying complexes and affect gene expression

(2009) Ahmad M. Khalil et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA

(2009) T. M. Wheeler et al.   SCIENCE

Polycomb Proteins Targeted by a Short Repeat RNA to the Mouse X Chromosome

(2008) J. Zhao et al.   SCIENCE