RNA-Based Therapeutics: From Antisense Oligonucleotides to miRNAs

Patisiran for the treatment of hereditary transthyretin-mediated amyloidosis

(2019) Junyi Yang   Expert Review of Clinical Pharmacology

RNA Biology Provides New Therapeutic Targets for Human Disease

(2019) Lorna W. Harries   Frontiers in Genetics

Re-Engineering RNA Molecules into Therapeutic Agents

(2019) Martin Egli et al.   ACCOUNTS OF CHEMICAL RESEARCH

Antiviral RNAi in Insects and Mammals: Parallels and Differences

(2019) Susan Schuster et al.   Viruses-Basel

Identifying causal variants and genes using functional genomics in specialized cell types and contexts

(2019) Boxiang Liu et al.   HUMAN GENETICS

MicroRNA-215-5p Treatment Suppresses Mesothelioma Progression via the MDM2-p53-Signaling Axis

(2019) Anand Singh et al.   MOLECULAR THERAPY

An Approach to Identify Individual Functional Single Nucleotide Polymorphisms and Isoform MicroRNAs

(2019) Ying Wang et al.   Biomed Research International

Micrornas in rheumatoid arthritis: From pathogenesis to clinical impact

(2019) Gerasimos Evangelatos et al.   AUTOIMMUNITY REVIEWS

miRNA Profiling for Early Detection and Treatment of Duchenne Muscular Dystrophy

(2019) Heather C. Hrach et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

MicroRNAs in acute pancreatitis: From pathogenesis to novel diagnosis and therapy

(2019) Yi Yang et al.   JOURNAL OF CELLULAR PHYSIOLOGY

The four‑microRNA signature identified by bioinformatics analysis predicts the prognosis of nasopharyngeal carcinoma patients

(2019) Siwei Zhang et al.   ONCOLOGY REPORTS

MicroRNA signature associated with treatment response in myasthenia gravis: A further step towards precision medicine

(2019) Paola Cavalcante et al.   PHARMACOLOGICAL RESEARCH

CRISPR: A Screener’s Guide

(2019) Carlos le Sage et al.   SLAS Discovery

Regulation of cardiac stem cells by microRNAs: State-of-the-art

(2019) Armita Mahdavi Gorabi et al.   BIOMEDICINE & PHARMACOTHERAPY

Family dynamics in transthyretin-related familial amyloid polyneuropathy Val30Met: does genetic risk affect family functioning?

(2018) Alice Lopes et al.   CLINICAL GENETICS

The genetic heterogeneity of hereditary transthyretin amyloidosis in a sample of the Brazilian population

(2018) Carolina Lavigne-Moreira et al.   JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM

Self-Delivering RNAi Targeting PD-1 Improves Tumor-Specific T Cell Functionality for Adoptive Cell Therapy of Malignant Melanoma

(2018) Maarten A. Ligtenberg et al.   MOLECULAR THERAPY

Oligonucleotide Drugs for Transthyretin Amyloidosis

(2018) Joel N. Buxbaum   NEW ENGLAND JOURNAL OF MEDICINE

Functional miRNAs in breast cancer drug resistance

(2018) Weizi Hu et al.   OncoTargets and Therapy

Hereditary transthyretin amyloidosis: baseline characteristics of patients in the NEURO-TTR trial

(2018) Marcia Waddington-Cruz et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Cobomarsen, an oligonucleotide inhibitor of miR-155, co-ordinately regulates multiple survival pathways to reduce cellular proliferation and survival in cutaneous T-cell lymphoma

(2018) Anita G. Seto et al.   BRITISH JOURNAL OF HAEMATOLOGY

Polypharmacology by Design: A Medicinal Chemist’s Perspective on Multitargeting Compounds

(2018) Ewgenij Proschak et al.   JOURNAL OF MEDICINAL CHEMISTRY

Circulating microRNA biomarkers for lung cancer detection in Western populations

(2018) Haixin Yu et al.   Cancer Medicine

An isomiR expression panel based novel breast cancer classification approach using improved mutual information

(2018) Chaowang Lan et al.   BMC Medical Genomics

A microRNA-29 mimic (remlarsen) represses extracellular matrix expression and fibroplasia in the skin

(2018) Corrie L. Gallant-Behm et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Nusinersen for the treatment of spinal muscular atrophy

(2017) Claudia A. Chiriboga   Expert Review of Neurotherapeutics

New treatments for serious conditions: ethical implications

(2017) N M P King et al.   GENE THERAPY

Targeting noncoding RNAs in disease

(2017) Brian D. Adams et al.   JOURNAL OF CLINICAL INVESTIGATION

CRISPR/Cas9 library screening for drug target discovery

(2017) Morito Kurata et al.   JOURNAL OF HUMAN GENETICS

Safety and activity of microRNA-loaded minicells in patients with recurrent malignant pleural mesothelioma: a first-in-man, phase 1, open-label, dose-escalation study

(2017) Nico van Zandwijk et al.   LANCET ONCOLOGY

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

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

Web-based NGS data analysis using miRMaster: a large-scale meta-analysis of human miRNAs

(2017) Tobias Fehlmann et al.   NUCLEIC ACIDS RESEARCH

Knowledge about the presence or absence of miRNA isoforms (isomiRs) can successfully discriminate amongst 32 TCGA cancer types

(2017) Aristeidis G. Telonis et al.   NUCLEIC ACIDS RESEARCH

Polymerase-free measurement of microRNA-122 with single base specificity using single molecule arrays: Detection of drug-induced liver injury

(2017) David M. Rissin et al.   PLoS One

FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

(2017) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

(2017) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

miRNA in Circulating Microvesicles as Biomarkers for Age-Related Cognitive Decline

(2017) Asha Rani et al.   Frontiers in Aging Neuroscience

Advances in the delivery of RNA therapeutics: from concept to clinical reality

(2017) James C. Kaczmarek et al.   Genome Medicine

Nusinersen for the treatment of spinal muscular atrophy

(2017) Claudia A. Chiriboga   Expert Review of Neurotherapeutics

As Technologies for Nucleotide Therapeutics Mature, Products Emerge

(2017) Jennifer M. Beierlein et al.   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

microRNA-guided diagnostics in clinical samples

(2016) Dakota Gustafson et al.   BEST PRACTICE & RESEARCH CLINICAL ENDOCRINOLOGY & METABOLISM

mRNA-based therapeutics–Advances and perspectives

(2016) O. V. Sergeeva et al.   BIOCHEMISTRY-MOSCOW

Is miR-34a a Well-equipped Swordsman to Conquer Temple of Molecular Oncology?

(2016) Ammad Ahmad Farooqi et al.   Chemical Biology & Drug Design

Non-coding RNAs as drug targets

(2016) Masayuki Matsui et al.   NATURE REVIEWS DRUG DISCOVERY

Prediction of human miRNA target genes using computationally reconstructed ancestral mammalian sequences

(2016) Mickael Leclercq et al.   NUCLEIC ACIDS RESEARCH

miRNA Deregulation in Cancer Cells and the Tumor Microenvironment

(2016) R. Rupaimoole et al.   Cancer Discovery

Empirical insights into the stochasticity of small RNA sequencing

(2016) Li-Xuan Qin et al.   Scientific Reports

An overview of microRNAs

(2015) Scott M. Hammond   ADVANCED DRUG DELIVERY REVIEWS

Exon skipping therapy for Duchenne muscular dystrophy

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

Evolving landscape in the management of transthyretin amyloidosis

(2015) Philip N. Hawkins et al.   ANNALS OF MEDICINE

Validated MicroRNA Target Databases: An Evaluation

(2015) Yun Ji Diana Lee et al.   DRUG DEVELOPMENT RESEARCH

The tumor-suppressive and potential therapeutic functions of miR-34a in epithelial carcinomas

(2015) Brian D. Adams et al.   EXPERT OPINION ON THERAPEUTIC TARGETS

PHASE 2 OPEN-LABEL EXTENSION STUDY OF PATISIRAN, AN INVESTIGATIONAL RNAI THERAPEUTIC FOR THE TREATMENT OF FAMILIAL AMYLOID POLYNEUROPATHY

(2015) David Adams et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

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

Multi-platform microRNA profiling of hepatoblastoma patients using formalin fixed paraffin embedded archival samples

(2015) Anna L. Leichter et al.   GigaScience

MicroRNA-targeting therapeutics for hepatitis C

(2014) Jihae Baek et al.   ARCHIVES OF PHARMACAL RESEARCH

Global population-specific variation in miRNA associated with cancer risk and clinical biomarkers

(2014) Renata A Rawlings-Goss et al.   BMC Medical Genomics

Development of microRNA therapeutics is coming of age

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

Current issues of RNAi therapeutics delivery and development

(2014) D. Haussecker   JOURNAL OF CONTROLLED RELEASE

CNS involvement in V30M transthyretin amyloidosis: clinical, neuropathological and biochemical findings

(2014) L. F. Maia et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Therapeutic targeting of microRNAs: current status and future challenges

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

Regulation of microRNA biogenesis

(2014) Minju Ha et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Mir-34: A New Weapon Against Cancer?

(2014) Gabriella Misso et al.   Molecular Therapy-Nucleic Acids

Restoring expression of miR-16: a novel approach to therapy for malignant pleural mesothelioma

(2013) G. Reid et al.   ANNALS OF ONCOLOGY

Molecular Mechanisms of RNA Interference

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

The let-7/LIN-41 Pathway Regulates Reprogramming to Human Induced Pluripotent Stem Cells by Controlling Expression of Prodifferentiation Genes

(2013) Kathleen A. Worringer et al.   Cell Stem Cell

Circulating miRNAs as sensitive and specific biomarkers for the diagnosis and monitoring of human diseases: Promises and challenges

(2013) V. De Guire et al.   CLINICAL BIOCHEMISTRY

First microRNA mimic enters clinic

(2013) Aaron Bouchie   NATURE BIOTECHNOLOGY

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

(2013) Teresa Coelho et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

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

RNA-Based Therapeutics: Current Progress and Future Prospects

(2012) John C. Burnett et al.   CHEMISTRY & BIOLOGY

RNA Interference for Viral Infections

(2012) Stephen J. Blake et al.   CURRENT DRUG TARGETS

Discovering the first microRNA-targeted drug

(2012) Morten Lindow et al.   JOURNAL OF CELL BIOLOGY

Short Structured RNAs with Low GC Content Are Selectively Lost during Extraction from a Small Number of Cells

(2012) Young-Kook Kim et al.   MOLECULAR CELL

Ophthalmic drug discovery: novel targets and mechanisms for retinal diseases and glaucoma

(2012) Kang Zhang et al.   NATURE REVIEWS DRUG DISCOVERY

Liver-specific microRNA-122: Biogenesis and function

(2012) Catherine Jopling   RNA Biology

IsomiRs – the overlooked repertoire in the dynamic microRNAome

(2012) Corine T. Neilsen et al.   TRENDS IN GENETICS

Minicells: Versatile vectors for targeted drug or si/shRNA cancer therapy

(2011) Jennifer A MacDiarmid et al.   CURRENT OPINION IN BIOTECHNOLOGY

Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72 400 specimens

(2011) Elaine A Sugarman et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Special delivery: targeted therapy with small RNAs

(2011) D Peer et al.   GENE THERAPY

Familial amyloid polyneuropathy

(2011) Violaine Planté-Bordeneuve et al.   LANCET NEUROLOGY

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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

Carrier-free cellular uptake and the gene-silencing activity of the lipophilic siRNAs is strongly affected by the length of the linker between siRNA and lipophilic group

(2011) Natalya S. Petrova et al.   NUCLEIC ACIDS RESEARCH

Subcellular Fate and Off-Target Effects of siRNA, shRNA, and miRNA

(2011) Saurabh Singh et al.   PHARMACEUTICAL RESEARCH

Delivery of siRNA Therapeutics: Barriers and Carriers

(2010) Jie Wang et al.   AAPS Journal

Thelet-7microRNA reduces tumor growth in mouse models of lung cancer

(2010) Aurora Esquela-Kerscher et al.   CELL CYCLE

Robust MicroRNA Stability in Degraded RNA Preparations from Human Tissue and Cell Samples

(2010) M. Jung et al.   CLINICAL CHEMISTRY

The MicroRNA Spectrum in 12 Body Fluids

(2010) J. A. Weber et al.   CLINICAL CHEMISTRY

The investigation of polymer-siRNA nanoparticle for gene therapy of gastric cancer in vitro

(2010) Wu   International Journal of Nanomedicine

Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver

(2010) Dirk Grimm et al.   JOURNAL OF CLINICAL INVESTIGATION

MicroRNA Networks Alter to Conform to Transcription Factor Networks Adding Redundancy and Reducing the Repertoire of Target Genes for Coordinated Regulation

(2010) H. Iwama et al.   MOLECULAR BIOLOGY AND EVOLUTION

LEDGF Hybrids Efficiently Retarget Lentiviral Integration Into Heterochromatin

(2010) Rik Gijsbers et al.   MOLECULAR THERAPY

Rational Design of Immunostimulatory siRNAs

(2010) Michael P Gantier et al.   MOLECULAR THERAPY

A Direct Comparison of Anti-microRNA Oligonucleotide Potency

(2010) Kim A. Lennox et al.   PHARMACEUTICAL RESEARCH

Systematic comparison of microarray profiling, real-time PCR, and next-generation sequencing technologies for measuring differential microRNA expression

(2010) A. Git et al.   RNA

RNA-Based Gene Therapy for HIV with Lentiviral Vector-Modified CD34+ Cells in Patients Undergoing Transplantation for AIDS-Related Lymphoma

(2010) D. L. DiGiusto et al.   Science Translational Medicine

RNAi and small interfering RNAs in human disease therapeutic applications

(2010) Monica R. Lares et al.   TRENDS IN BIOTECHNOLOGY

Anti-tumor effect of small interfering RNA targeting the androgen receptor in human androgen-independent prostate cancer cells

(2009) Koji Azuma et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

MicroRNAs: Target Recognition and Regulatory Functions

(2009) David P. Bartel   CELL

Exploring Chemical Modifications for siRNA Therapeutics: A Structural and Functional Outlook

(2009) Siddharth Shukla et al.   ChemMedChem

Redefining MicroRNA Targets

(2009) Hervé Seitz   CURRENT BIOLOGY

The role of RNAi and microRNAs in animal virus replication and antiviral immunity

(2009) J. L. Umbach et al.   GENES & DEVELOPMENT

DNA as therapeutics; an update

(2009) P Saraswat et al.   INDIAN JOURNAL OF PHARMACEUTICAL SCIENCES

MicroRNAs in clinical oncology: at the crossroads between promises and problems

(2009) S M Metias et al.   JOURNAL OF CLINICAL PATHOLOGY

miR-24 Inhibits Cell Proliferation by Targeting E2F2, MYC, and Other Cell-Cycle Genes via Binding to “Seedless” 3′UTR MicroRNA Recognition Elements

(2009) Ashish Lal et al.   MOLECULAR CELL

Combinatorial RNAi Against HIV-1 Using Extended Short Hairpin RNAs

(2009) Ying Poi Liu et al.   MOLECULAR THERAPY

Low-level shRNA Cytotoxicity Can Contribute to MYC-induced Hepatocellular Carcinoma in Adult Mice

(2009) Shelly Beer et al.   MOLECULAR THERAPY

Sequential treatment of drug-resistant tumors with targeted minicells containing siRNA or a cytotoxic drug

(2009) Jennifer A MacDiarmid et al.   NATURE BIOTECHNOLOGY

Systemic administration of optimized aptamer-siRNA chimeras promotes regression of PSMA-expressing tumors

(2009) Justin P Dassie et al.   NATURE BIOTECHNOLOGY

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

(2009) Aimee L. Jackson et al.   NATURE REVIEWS DRUG DISCOVERY

Selection, characterization and application of new RNA HIV gp 120 aptamers for facile delivery of Dicer substrate siRNAs into HIV infected cells

(2009) Jiehua Zhou et al.   NUCLEIC ACIDS RESEARCH

Intra-Platform Repeatability and Inter-Platform Comparability of MicroRNA Microarray Technology

(2009) Fumiaki Sato et al.   PLoS One

COMBINATION THERAPY USING THE SMALL INTERFERING RNA BEVASIRANIB

(2009) LAWRENCE SINGERMAN   RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES

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

(2009) R. E. Lanford et al.   SCIENCE

Tumor Regression in Mice by Delivery of Bcl-2 Small Interfering RNA with Pegylated Cationic Liposomes

(2008) Satoru Sonoke et al.   CANCER RESEARCH

T Cell-Specific siRNA Delivery Suppresses HIV-1 Infection in Humanized Mice

(2008) Priti Kumar et al.   CELL

Position-Dependent Function for a Tandem MicroRNA miR-122-Binding Site Located in the Hepatitis C Virus RNA Genome

(2008) Catherine L. Jopling et al.   Cell Host & Microbe

Characterization of microRNAs in serum: a novel class of biomarkers for diagnosis of cancer and other diseases

(2008) Xi Chen et al.   CELL RESEARCH

Prevention of interferon-stimulated gene expression using microRNA-designed hairpins

(2008) M Bauer et al.   GENE THERAPY

Engineering and optimization of the miR-106b cluster for ectopic expression of multiplexed anti-HIV RNAs

(2008) L A Aagaard et al.   GENE THERAPY

Most mammalian mRNAs are conserved targets of microRNAs

(2008) R. C. Friedman et al.   GENOME RESEARCH

VEGF siRNA Delivery System Using Arginine-Grafted Bioreducible Poly(disulfide amine)

(2008) Sun Hwa Kim et al.   MOLECULAR PHARMACEUTICS

Artificial MicroRNAs as siRNA Shuttles: Improved Safety as Compared to shRNAs In vitro and In vivo

(2008) Ryan L Boudreau et al.   MOLECULAR THERAPY

LNA-mediated microRNA silencing in non-human primates

(2008) Joacim Elmén et al.   NATURE

5′-triphosphate-siRNA: turning gene silencing and Rig-I activation against melanoma

(2008) Hendrik Poeck et al.   NATURE MEDICINE

The miR-15a–miR-16-1 cluster controls prostate cancer by targeting multiple oncogenic activities

(2008) Désirée Bonci et al.   NATURE MEDICINE

Inhibition of HIV-1 by multiple siRNAs expressed from a single microRNA polycistron

(2008) Ying Poi Liu et al.   NUCLEIC ACIDS RESEARCH

Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

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

Suppression of non-small cell lung tumor development by the let-7 microRNA family

(2008) M. S. Kumar et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Coexpression of Argonaute-2 enhances RNA interference toward perfect match binding sites

(2008) S. Diederichs et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates

(2008) M. Frank-Kamenetsky et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Circulating microRNAs as stable blood-based markers for cancer detection

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

Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs

(2008) R. L. Boudreau et al.   RNA

Systemic Leukocyte-Directed siRNA Delivery Revealing Cyclin D1 as an Anti-Inflammatory Target

(2008) D. Peer et al.   SCIENCE