Silencing disease genes in the laboratory and the clinic

siRNA-induced immunostimulation through TLR7 promotes antitumoral activity against HPV-driven tumors in vivo

(2011) Norliana Khairuddin et al.   IMMUNOLOGY AND CELL BIOLOGY

RNAi momentum fizzles as pharma shifts priorities

(2011) Charles Schmidt   NATURE BIOTECHNOLOGY

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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

Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes

(2011) Erich Koller et al.   NUCLEIC ACIDS RESEARCH

Potent and systematic RNAi mediated silencing with single oligonucleotide compounds

(2011) J. Lapierre et al.   RNA

Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy

(2011) Aurélie Goyenvalle et al.   Skeletal Muscle

Delivery of siRNA Therapeutics: Barriers and Carriers

(2010) Jie Wang et al.   AAPS Journal

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

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

Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne muscular dystrophy

(2010) Hong M. Moulton et al.   BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES

Clinical status of duplex RNA

(2010) Jonathan K. Watts et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

The Telomerase Inhibitor Imetelstat Depletes Cancer Stem Cells in Breast and Pancreatic Cancer Cell Lines

(2010) I. Joseph et al.   CANCER RESEARCH

Targeting the Cytoprotective Chaperone, Clusterin, for Treatment of Advanced Cancer

(2010) A. Zoubeidi et al.   CLINICAL CANCER RESEARCH

Down-modulation of cancer targets using locked nucleic acid (LNA)-based antisense oligonucleotides without transfection

(2010) Y Zhang et al.   GENE 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

Therapeutic Advances in MicroRNA Targeting

(2010) Rusty L Montgomery et al.   JOURNAL OF CARDIOVASCULAR PHARMACOLOGY

miRNAs in human cancer

(2010) Thalia A Farazi et al.   JOURNAL OF PATHOLOGY

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

Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO

(2010) HaiFang Yin et al.   MOLECULAR THERAPY

Targeted Delivery of siRNA to Macrophages for Anti-inflammatory Treatment

(2010) Sang-Soo Kim et al.   MOLECULAR THERAPY

Rational Design of Immunostimulatory siRNAs

(2010) Michael P Gantier et al.   MOLECULAR THERAPY

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

(2010) Mark E. Davis et al.   NATURE

Safety signal dampens reception for mipomersen antisense

(2010) Jim Kling   NATURE BIOTECHNOLOGY

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Targeting microRNAs in cancer: rationale, strategies and challenges

(2010) Ramiro Garzon et al.   NATURE REVIEWS DRUG DISCOVERY

Anti-tumor activity of splice-switching oligonucleotides

(2010) John A. Bauman et al.   NUCLEIC ACIDS RESEARCH

Synergistic effects between analogs of DNA and RNA improve the potency of siRNA-mediated gene silencing

(2010) Glen F. Deleavey et al.   NUCLEIC ACIDS RESEARCH

Short locked nucleic acid antisense oligonucleotides potently reduce apolipoprotein B mRNA and serum cholesterol in mice and non-human primates

(2010) Ellen Marie Straarup et al.   NUCLEIC ACIDS RESEARCH

Remembering Paul C. Zamecnik, M.D., ”Father of Antisense” (1912–2009)

(2010) Sudhir Agrawal   OLIGONUCLEOTIDES

Lipid-based systemic delivery of siRNA

(2009) Yu-Cheng Tseng et al.   ADVANCED DRUG DELIVERY REVIEWS

RNA Interference-Mediated Silencing of the Respiratory Syncytial Virus Nucleocapsid Defines a Potent Antiviral Strategy

(2009) R. Alvarez et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

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

(2009) Natee Jearawiriyapaisarn et al.   CARDIOVASCULAR RESEARCH

A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice

(2009) HaiFang Yin et al.   HUMAN MOLECULAR GENETICS

Confirming the RNAi-mediated mechanism of action of siRNA-based cancer therapeutics in mice

(2009) Adam D. Judge et al.   JOURNAL OF CLINICAL INVESTIGATION

Less is more: therapeutic exon skipping for Duchenne muscular dystrophy

(2009) Annemieke Aartsma-Rus et al.   LANCET NEUROLOGY

Asymmetric Shorter-duplex siRNA Structures Trigger Efficient Gene Silencing With Reduced Nonspecific Effects

(2009) Chan Il Chang et al.   MOLECULAR THERAPY

On the road to reading the RNA-interference code

(2009) Haruhiko Siomi et al.   NATURE

In vivo delivery of siRNA to immune cells by conjugation to a TLR9 agonist enhances antitumor immune responses

(2009) Marcin Kortylewski 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

Efficient siRNA delivery into primary cells by a peptide transduction domain–dsRNA binding domain fusion protein

(2009) Akiko Eguchi et al.   NATURE BIOTECHNOLOGY

Erratum: Corrigendum: Transfection of small RNAs globally perturbs gene regulation by endogenous microRNAs

(2009) Aly A Khan et al.   NATURE BIOTECHNOLOGY

Knocking down barriers: advances in siRNA delivery

(2009) Kathryn A. Whitehead et al.   NATURE REVIEWS DRUG DISCOVERY

A rapid and sensitive method to detect siRNA-mediated mRNA cleavage in vivo using 5′ RACE and a molecular beacon probe

(2009) Annette Lasham et al.   NUCLEIC ACIDS RESEARCH

Sequence-non-specific effects of RNA interference triggers and microRNA regulators

(2009) Marta Olejniczak et al.   NUCLEIC ACIDS RESEARCH

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

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

A large-scale chemical modification screen identifies design rules to generate siRNAs with high activity, high stability and low toxicity

(2009) Jesper B. Bramsen et al.   NUCLEIC ACIDS RESEARCH

Therapeutic Potential of Splice-Switching Oligonucleotides

(2009) John Bauman et al.   OLIGONUCLEOTIDES

siRNA and Innate Immunity

(2009) Marjorie Robbins et al.   OLIGONUCLEOTIDES

Therapeutic Applications of DNA and RNA Aptamers

(2009) Kristina W. Thiel et al.   OLIGONUCLEOTIDES

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

(2009) R. E. Lanford et al.   SCIENCE

A Primer for Morpholino Use in Zebrafish

(2009) Brent R. Bill et al.   Zebrafish

2′F-Arabinonucleic acids (2′F-ANA) — History, properties, and new frontiers

(2008) Jonathan K Watts et al.   CANADIAN JOURNAL OF CHEMISTRY

Chemically modified siRNA: tools and applications

(2008) J WATTS et al.   DRUG DISCOVERY TODAY

The Business of RNAi Therapeutics

(2008) Dirk Haussecker   HUMAN GENE THERAPY

Misinterpreting the Therapeutic Effects of Small Interfering RNA Caused by Immune Stimulation

(2008) Marjorie Robbins et al.   HUMAN GENE THERAPY

Overcoming the Innate Immune Response to Small Interfering RNA

(2008) Adam Judge et al.   HUMAN GENE THERAPY

Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function

(2008) HaiFang Yin et al.   HUMAN MOLECULAR GENETICS

Modification of HER2 pre-mRNA alternative splicing and its effects on breast cancer cells

(2008) Jing Wan et al.   INTERNATIONAL JOURNAL OF CANCER

miR-122, a paradigm for the role of microRNAs in the liver

(2008) Muriel Girard et al.   JOURNAL OF HEPATOLOGY

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

(2008) Natee Jearawiriyapaisarn et al.   MOLECULAR THERAPY

Sequence- and target-independent angiogenesis suppression by siRNA via TLR3

(2008) Mark E. Kleinman et al.   NATURE

LNA-mediated microRNA silencing in non-human primates

(2008) Joacim Elmén et al.   NATURE

Asymmetric RNA duplexes mediate RNA interference in mammalian cells

(2008) Xiangao Sun et al.   NATURE BIOTECHNOLOGY

Resolution of liver cirrhosis using vitamin A–coupled liposomes to deliver siRNA against a collagen-specific chaperone

(2008) Yasushi Sato et al.   NATURE BIOTECHNOLOGY

Deep surveying of alternative splicing complexity in the human transcriptome by high-throughput sequencing

(2008) Qun Pan et al.   NATURE GENETICS

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

(2008) Hendrik Poeck et al.   NATURE MEDICINE

Potent inhibition of microRNA in vivo without degradation

(2008) Scott Davis et al.   NUCLEIC ACIDS RESEARCH

Mechanisms and strategies for effective delivery of antisense and siRNA oligonucleotides

(2008) R. Juliano et al.   NUCLEIC ACIDS RESEARCH

Effect of asymmetric terminal structures of short RNA duplexes on the RNA interference activity and strand selection

(2008) Masayuki Sano et al.   NUCLEIC ACIDS RESEARCH

Functional dissection of siRNA sequence by systematic DNA substitution: modified siRNA with a DNA seed arm is a powerful tool for mammalian gene silencing with significantly reduced off-target effect

(2008) Kumiko Ui-Tei et al.   NUCLEIC ACIDS RESEARCH

miR2Disease: a manually curated database for microRNA deregulation in human disease

(2008) Q. Jiang et al.   NUCLEIC ACIDS RESEARCH

Chemical Modification Patterns Compatible with High Potency Dicer-Substrate Small Interfering RNAs

(2008) Michael A. Collingwood et al.   OLIGONUCLEOTIDES

An Analysis of Human MicroRNA and Disease Associations

(2008) Ming Lu et al.   PLoS One

Effects of local mRNA structure on posttranscriptional gene silencing

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

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 microRNA with antisense oligonucleotides

(2007) Christine C. Esau   METHODS