Clinical Applications of Short Non-Coding RNA-Based Therapies in the Era of Precision Medicine

Lumasiran: First Approval

(2021) Lesley J. Scott et al.   DRUGS

Injectable hydrogel with MSNs/microRNA-21-5p delivery enables both immunomodification and enhanced angiogenesis for myocardial infarction therapy in pigs

(2021) Yan Li et al.   Science Advances

Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1

(2021) Yaacov Frishberg et al.   Clinical Journal of the American Society of Nephrology

Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

(2021) Sander F. Garrelfs et al.   NEW ENGLAND JOURNAL OF MEDICINE

microRNA ‐based diagnostic and therapeutic applications in cancer medicine

(2021) Lorenzo F. Sempere et al.   Wiley Interdisciplinary Reviews-RNA

Teprasiran, A Small Interfering RNA, for the Prevention of Acute Kidney Injury in High-Risk Patients Undergoing Cardiac Surgery: A Randomized Clinical Study

(2021) Matthias Thielmann et al.   CIRCULATION

The tangled history of mRNA vaccines

(2021) Elie Dolgin   NATURE

The current landscape of nucleic acid therapeutics

(2021) Jayesh A. Kulkarni et al.   Nature Nanotechnology

Stromal Modulation and Treatment of Metastatic Pancreatic Cancer with Local Intraperitoneal Triple miRNA/siRNA Nanotherapy

(2020) Ying Xie et al.   ACS Nano

Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR)

(2020) Daniel P. Judge et al.   CARDIOVASCULAR DRUGS AND THERAPY

Phase 1 study of MRX34, a liposomal miR-34a mimic, in patients with advanced solid tumours

(2020) David S. Hong et al.   BRITISH JOURNAL OF CANCER

Loss of microRNA ‐21 leads to profound stromal remodeling and short survival in K‐Ras ‐driven mouse models of pancreatic cancer

(2020) Josh Schipper et al.   INTERNATIONAL JOURNAL OF CANCER

AntimiR-21 Prevents Myocardial Dysfunction in a Pig Model of Ischemia/Reperfusion Injury

(2020) Rabea Hinkel et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Two Phase 3 Trials of Inclisiran in Patients with Elevated LDL Cholesterol

(2020) Kausik K. Ray et al.   NEW ENGLAND JOURNAL OF MEDICINE

Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia

(2020) Frederick J. Raal et al.   NEW ENGLAND JOURNAL OF MEDICINE

The powerful world of antisense oligonucleotides: From bench to bedside

(2020) Anaïs M. Quemener et al.   Wiley Interdisciplinary Reviews-RNA

Pancreatic cancer

(2020) Jonathan D Mizrahi et al.   LANCET

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

(2020) Alexandre J. Debacker et al.   MOLECULAR THERAPY

Advances in oligonucleotide drug delivery

(2020) Thomas C. Roberts et al.   NATURE REVIEWS DRUG DISCOVERY

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

(2020) Manisha Balwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Efficiency and Target Derepression of Anti-miR-92a: Results of a First in Human Study

(2020) Wesley Tyler Abplanalp et al.   Nucleic Acid Therapeutics

The Promising Role of miR-21 as a Cancer Biomarker and Its Importance in RNA-Based Therapeutics

(2020) Diana Bautista-Sánchez et al.   Molecular Therapy-Nucleic Acids

A Comprehensive Review of Cancer MicroRNA Therapeutic Delivery Strategies

(2020) Alexis Forterre et al.   Cancers

S1169 Twelve-Month Interim Analysis of Efficacy and Safety of Givosiran, an Investigational RNAi Therapeutic for Acute Hepatic Porphyria, in the ENVISION Open Label Extension

(2020) Eliane Sardh et al.   AMERICAN JOURNAL OF GASTROENTEROLOGY

Cobomarsen, an Oligonucleotide Inhibitor of miR-155, Slows DLBCL Tumor Cell Growth In Vitro and In Vivo

(2020) Eleni Anastasiadou et al.   CLINICAL CANCER RESEARCH

MicroRNAs and Long Non-Coding RNAs and Their Hormone-Like Activities in Cancer

(2019) Barbara Pardini et al.   Cancers

Dysregulated Expression of microRNA-21 and Disease Related Genes in Human Patients and Mouse Model of Alport Syndrome

(2019) Jifan Guo et al.   HUMAN GENE THERAPY

Reduction in urinary oxalate excretion in mouse models of Primary Hyperoxaluria by RNA interference inhibition of liver lactate dehydrogenase activity

(2019) Kyle D. Wood et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Treatment of cardiac transthyretin amyloidosis: an update

(2019) Michele Emdin et al.   EUROPEAN HEART JOURNAL

MP12-14 SAFETY AND EFFICACY STUDY OF LUMASIRAN, AN INVESTIGATIONAL RNA INTERFERENCE (RNAI) THERAPEUTIC, IN ADULT AND PEDIATRIC PATIENTS WITH PRIMARY HYPEROXALURIA TYPE 1 (PH1)

(2019) Yaacov Frishberg et al.   JOURNAL OF UROLOGY

The Potential for microRNA Therapeutics and Clinical Research

(2019) Johora Hanna et al.   Frontiers in Genetics

A Fit-for-Purpose Method for the Detection of Human Antibodies to Surface-Exposed Components of BMS-986263, a Lipid Nanoparticle-Based Drug Product Containing a siRNA Drug Substance

(2019) Uma Kavita et al.   AAPS Journal

MicroRNA-301a promotes pancreatic cancer invasion and metastasis through the JAK/STAT3 signaling pathway by targeting SOCS5

(2019) Hui Hu et al.   CARCINOGENESIS

Bone Marrow Mesenchymal Stem Cell-Derived Exosomal MicroRNA-126-3p Inhibits Pancreatic Cancer Development by Targeting ADAM9

(2019) Dong-Mei Wu et al.   Molecular Therapy-Nucleic Acids

Tumor penetrating nanomedicine targeting both an oncomiR and an oncogene in pancreatic cancer

(2019) Maud-Emmanuelle Gilles et al.   Oncotarget

RNA Interference Therapy with ARC‐520 Results in Prolonged HBsAg Response in Patients with Chronic Hepatitis B Infection

(2019) Man‐Fung Yuen et al.   HEPATOLOGY

Effects of Renal Impairment on the Pharmacokinetics, Efficacy, and Safety of Inclisiran: An Analysis of the ORION-7 and ORION-1 Studies

(2019) R. Scott Wright et al.   MAYO CLINIC PROCEEDINGS

Exosomal miRNA‐1231 derived from bone marrow mesenchymal stem cells inhibits the activity of pancreatic cancer

(2019) Song Shang et al.   Cancer Medicine

PCSK9-gene-silencing, cholesterol-lowering drug impresses

(2019) Cormac Sheridan   NATURE BIOTECHNOLOGY

MicroRNA-710 regulates multiple pathways of carcinogenesis in murine metastatic breast cancer

(2019) Byunghee Yoo et al.   PLoS One

Metazoan MicroRNAs

(2018) David P. Bartel   CELL

RNA-Targeted Therapeutics

(2018) Stanley T. Crooke et al.   Cell Metabolism

Hepatic-targeted RNA interference provides robust and persistent knockdown of alpha-1 antitrypsin levels in ZZ patients

(2018) Alice M. Turner et al.   JOURNAL OF HEPATOLOGY

Clinical utility of circulating non-coding RNAs — an update

(2018) Simone Anfossi et al.   Nature Reviews Clinical Oncology

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics

(2018) Aaron D. Springer et al.   Nucleic Acid Therapeutics

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

Regulation of microRNA function in animals

(2018) Luca F. R. Gebert et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

A synthetic microRNA-92a inhibitor (MRG-110) accelerates angiogenesis and wound healing in diabetic and non-diabetic wounds

(2018) Corrie L. Gallant-Behm et al.   WOUND REPAIR AND REGENERATION

miRBase: from microRNA sequences to function

(2018) Ana Kozomara et al.   NUCLEIC ACIDS RESEARCH

Exosomes derived from human umbilical cord mesenchymal stromal cells deliver exogenous miR-145-5p to inhibit pancreatic ductal adenocarcinoma progression

(2018) Yixuan Ding et al.   CANCER LETTERS

Preclinical Mammalian Safety Studies of EPHARNA (DOPC Nanoliposomal EphA2-Targeted siRNA)

(2017) Michael J. Wagner et al.   MOLECULAR CANCER THERAPEUTICS

Exosomes facilitate therapeutic targeting of oncogenic KRAS in pancreatic cancer

(2017) Sushrut Kamerkar et al.   NATURE

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

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

Inclisiran in Patients at High Cardiovascular Risk with Elevated LDL Cholesterol

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

Molecular Mechanisms of Antisense Oligonucleotides

(2017) Stanley T. Crooke   Nucleic Acid Therapeutics

Therapy targeted to the metastatic niche is effective in a model of stage IV breast cancer

(2017) Byunghee Yoo et al.   Scientific Reports

Phase I study of MRX34, a liposomal miR-34a mimic, administered twice weekly in patients with advanced solid tumors

(2016) Muhammad S. Beg et al.   INVESTIGATIONAL NEW DRUGS

The biology and function of exosomes in cancer

(2016) Raghu Kalluri   JOURNAL OF CLINICAL INVESTIGATION

Functional analysis of microRNA-122 binding sequences of hepatitis C virus and identification of variants with high resistance against a specific antagomir

(2016) Yi-Ping Li et al.   JOURNAL OF GENERAL VIROLOGY

ALN-RSV01 for prevention of bronchiolitis obliterans syndrome after respiratory syncytial virus infection in lung transplant recipients

(2016) Jens Gottlieb et al.   JOURNAL OF HEART AND LUNG TRANSPLANTATION

Preclinical Safety Evaluation in Rats of a Polymeric Matrix Containing an siRNA Drug Used as a Local and Prolonged Delivery System for Pancreatic Cancer Therapy

(2016) Yuval Ramot et al.   TOXICOLOGIC PATHOLOGY

Clinical development of TargomiRs, a miRNA mimic-based treatment for patients with recurrent thoracic cancer

(2016) Glen Reid et al.   Epigenomics

Pancreatic cancer

(2016) Jorg Kleeff et al.   Nature Reviews Disease Primers

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

A Significant Metabolic and Radiological Response after a Novel Targeted MicroRNA-based Treatment Approach in Malignant Pleural Mesothelioma

(2015) Steven C. Kao et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Combining miR-10b-Targeted Nanotherapy with Low-Dose Doxorubicin Elicits Durable Regressions of Metastatic Breast Cancer

(2015) B. Yoo et al.   CANCER RESEARCH

Technologies for controlled, local delivery of siRNA

(2015) Samantha M. Sarett et al.   JOURNAL OF CONTROLLED RELEASE

RNAi therapy targeting KRAS in combination with chemotherapy for locally advanced pancreatic cancer patients

(2015) Talia Golan et al.   Oncotarget

Turning 21: Induction of miR-21 as a Key Switch in the Inflammatory Response

(2015) Frederick J. Sheedy   Frontiers in Immunology

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

Epidemiology and Outcomes in Community-Acquired Versus Hospital-Acquired AKI

(2014) A. Wonnacott et al.   Clinical Journal of the American Society of Nephrology

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

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

Argonaute proteins: functional insights and emerging roles

(2013) Gunter Meister   NATURE REVIEWS GENETICS

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

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

Discovering the first microRNA-targeted drug

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

Essential metabolic, anti-inflammatory, and anti-tumorigenic functions of miR-122 in liver

(2012) Shu-hao Hsu et al.   JOURNAL OF CLINICAL INVESTIGATION

RNA Interference, RSV, and Lung Transplantation

(2011) Todd L. Astor   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

RNAi-Based Treatment for Neovascular Age-Related Macular Degeneration by Sirna-027

(2010) Peter K. Kaiser et al.   AMERICAN JOURNAL OF OPHTHALMOLOGY

Development of Quantitative Molecular Clinical End Points for siRNA Clinical Trials

(2010) Robyn P. Hickerson et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Glomerular filtration rate, proteinuria, and the incidence and consequences of acute kidney injury: a cohort study

(2010) Matthew T James et al.   LANCET

Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles

(2010) Mark E. Davis et al.   NATURE

A randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus

(2010) J. DeVincenzo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

First-in-human Mutation-targeted siRNA Phase Ib Trial of an Inherited Skin Disorder

(2009) Sancy A Leachman et al.   MOLECULAR THERAPY

Evaluation of the safety, tolerability and pharmacokinetics of ALN-RSV01, a novel RNAi antiviral therapeutic directed against respiratory syncytial virus (RSV)

(2008) J DEVINCENZO et al.   ANTIVIRAL RESEARCH

miR-29 miRNAs activate p53 by targeting p85α and CDC42

(2008) Seong-Yeon Park et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Impact of tumor-specific targeting and dosing schedule on tumor growth inhibition after intravenous administration of siRNA-containing nanoparticles

(2007) Derek W. Bartlett et al.   BIOTECHNOLOGY AND BIOENGINEERING