RNA therapeutics: beyond RNA interference and antisense oligonucleotides

Correction of β654-thalassaemia mice using direct intravenous injection of siRNA and antisense RNA vectors

(2011) Shu-Yang Xie et al.   INTERNATIONAL JOURNAL OF HEMATOLOGY

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

Alternative for Anti-TNF Antibodies for Arthritis Treatment

(2011) Joseph Paquet et al.   MOLECULAR THERAPY

Stabilization of Expanded (CTG)•(CAG) Repeats by Antisense Oligonucleotides

(2011) Masayuki Nakamori et al.   MOLECULAR THERAPY

Is RNAi Dead?

(2011) Arthur M Krieg   MOLECULAR THERAPY

Pip5 Transduction Peptides Direct High Efficiency Oligonucleotide-mediated Dystrophin Exon Skipping in Heart and Phenotypic Correction in mdx Mice

(2011) HaiFang Yin et al.   MOLECULAR THERAPY

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

(2011) Yimin Hua et al.   NATURE

Targeting RNA to treat neuromuscular disease

(2011) Francesco Muntoni et al.   NATURE REVIEWS DRUG DISCOVERY

Current prospects for RNA interference-based therapies

(2011) Beverly L. Davidson et al.   NATURE REVIEWS GENETICS

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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

Basic peptide-morpholino oligomer conjugate that is very effective in killing bacteria by gene-specific and nonspecific modes

(2011) D. Wesolowski et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

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

Reduction of herpes simplex virus type-2 replication in cell cultures and in rodent models with peptide-conjugated morpholino oligomers

(2010) Kathleen Eide et al.   ANTIVIRAL THERAPY

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

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

Beta-thalassemia

(2010) Antonio Cao et al.   GENETICS IN MEDICINE

Safety Pharmacology and Genotoxicity Evaluation of AVI-4658

(2010) Peter Sazani et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

Engineered External Guide Sequences Effectively Block Viral Gene Expression and Replication in Cultured Cells

(2010) Xiaohong Jiang et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Randomized Phase II Study of Docetaxel and Prednisone With or Without OGX-011 in Patients With Metastatic Castration-Resistant Prostate Cancer

(2010) Kim N. Chi et al.   JOURNAL OF CLINICAL ONCOLOGY

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

Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model

(2010) Hans Heemskerk et al.   MOLECULAR THERAPY

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Advanced antisense therapies for postexposure protection against lethal filovirus infections

(2010) Travis K Warren et al.   NATURE MEDICINE

RNA sequencing: advances, challenges and opportunities

(2010) Fatih Ozsolak et al.   NATURE REVIEWS GENETICS

Roche Exits RNAi Field, Cuts 4800 Jobs

(2010) J. Couzin-Frankel   SCIENCE

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

Pathogenic mechanisms of myotonic dystrophy

(2009) Johanna E. Lee et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

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

(2009) Natee Jearawiriyapaisarn et al.   CARDIOVASCULAR RESEARCH

RNA-Based Therapeutics: Ready for Delivery?

(2009) Laura Bonetta   CELL

Phase II Trial of OGX-011 in Combination with Docetaxel in Metastatic Breast Cancer

(2009) S. Chia et al.   CLINICAL CANCER RESEARCH

LDH correlation with survival in advanced melanoma from two large, randomised trials (Oblimersen GM301 and EORTC 18951)

(2009) Sanjiv S. Agarwala et al.   EUROPEAN JOURNAL OF CANCER

Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino

(2009) B Wu et al.   GENE THERAPY

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

(2009) Annemieke Aartsma-Rus et al.   HUMAN MUTATION

Cationic phosphorodiamidate morpholino oligomers efficiently prevent growth of Escherichia coli in vitro and in vivo

(2009) Brett L. Mellbye et al.   JOURNAL OF ANTIMICROBIAL CHEMOTHERAPY

In vivocomparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

(2009) Hans A. Heemskerk et al.   JOURNAL OF GENE MEDICINE

Oligonucleotide-Mediated Survival of Motor Neuron Protein Expression in CNS Improves Phenotype in a Mouse Model of Spinal Muscular Atrophy

(2009) J. H. Williams et al.   JOURNAL OF NEUROSCIENCE

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

(2009) Maria Kinali et al.   LANCET NEUROLOGY

Phase III randomised study of dexamethasone with or without oblimersen sodium for patients with advanced multiple myeloma

(2009) Asher A. Chanan-Khan et al.   LEUKEMIA & LYMPHOMA

Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping

(2009) Aurélie Goyenvalle et al.   MOLECULAR THERAPY

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

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

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice

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

Inactivation of expression of several genes in a variety of bacterial species by EGS technology

(2009) N. Shen et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

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

(2009) T. M. Wheeler et al.   SCIENCE

Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice

(2008) Yimin Hua et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Variations in Amino Acid Composition of Antisense Peptide-Phosphorodiamidate Morpholino Oligomer Affect Potency against Escherichia coli In Vitro and In Vivo

(2008) B. L. Mellbye et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

A Morpholino Oligomer Targeting Highly Conserved Internal Ribosome Entry Site Sequence Is Able To Inhibit Multiple Species of Picornavirus

(2008) J. K. Stone et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

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

Inhibition of influenza A H3N8 virus infections in mice by morpholino oligomers

(2008) Christopher Lupfer et al.   ARCHIVES OF VIROLOGY

Alternative splicing and disease

(2008) Jamal Tazi et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Chemically modified siRNA: tools and applications

(2008) J WATTS et al.   DRUG DISCOVERY TODAY

Morpholino oligomers targeting the PB1 and NP genes enhance the survival of mice infected with highly pathogenic influenza A H7N7 virus

(2008) G. Gabriel et al.   JOURNAL OF GENERAL VIROLOGY

An Endogenous TNF-α Antagonist Induced by Splice-switching Oligonucleotides Reduces Inflammation in Hepatitis and Arthritis Mouse Models

(2008) Maria A Graziewicz et al.   MOLECULAR THERAPY

Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of mdx Mice

(2008) Natee Jearawiriyapaisarn et al.   MOLECULAR THERAPY

Inhibition of Respiratory Syncytial Virus Infections With Morpholino Oligomers in Cell Cultures and in Mice

(2008) Shen-Hao Lai et al.   MOLECULAR THERAPY

Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer

(2008) B. Wu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Inhibition of alphavirus infection in cell culture and in mice with antisense morpholino oligomers

(2008) Slobodan Paessler et al.   VIROLOGY