Oligonucleotide therapeutics: chemistry, delivery and clinical progress

siRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in Hepatocytes

(2015) Shigeo Matsuda et al.   ACS Chemical Biology

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

(2015) Richard S. Geary et al.   ADVANCED DRUG DELIVERY REVIEWS

Preclinical and clinical development of siRNA-based therapeutics

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

Efficient Synthesis and Biological Evaluation of 5′-GalNAc Conjugated Antisense Oligonucleotides

(2015) Michael E. Østergaard et al.   BIOCONJUGATE CHEMISTRY

Hepatocyte-Specific Delivery of siRNAs Conjugated to Novel Non-nucleosidic TrivalentN-Acetylgalactosamine Elicits Robust Gene Silencing in Vivo

(2015) Kallanthottathil G. Rajeev et al.   CHEMBIOCHEM

Synthetic Nucleic Acid Delivery Systems: Present and Perspectives

(2015) Bogdan Draghici et al.   JOURNAL OF MEDICINAL CHEMISTRY

Synthesis and Properties of 2′-Deoxy-2′,4′-difluoroarabinose-Modified Nucleic Acids

(2015) Saúl Martínez-Montero et al.   JOURNAL OF ORGANIC CHEMISTRY

Advances in CRISPR-Cas9 genome engineering: lessons learned from RNA interference

(2015) Rodolphe Barrangou et al.   NUCLEIC ACIDS RESEARCH

C5-azobenzene-functionalized locked nucleic acid uridine: isomerization properties, hybridization ability, and enzymatic stability

(2015) K. Morihiro et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Immunomodulatory spherical nucleic acids

(2015) Aleksandar F. Radovic-Moreno et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Chimeric Antisense Oligonucleotide Conjugated to α-Tocopherol

(2015) Tomoko Nishina et al.   Molecular Therapy-Nucleic Acids

Allele-Selective Inhibition of Mutant Atrophin-1 Expression by Duplex and Single-Stranded RNAs

(2014) Jiaxin Hu et al.   BIOCHEMISTRY

C5-Amino acid functionalized LNA: positively poised for antisense applications

(2014) Dale C. Guenther et al.   CHEMICAL COMMUNICATIONS

Nucleic Acid Aptamer-Mediated Drug Delivery for Targeted Cancer Therapy

(2014) Huijie Zhu et al.   ChemMedChem

Aptamer-based therapeutics of the past, present and future: from the perspective of eye-related diseases

(2014) Jagat R. Kanwar et al.   DRUG DISCOVERY TODAY

Oligonucleotide delivery: a patent review (2010 – 2013)

(2014) Santiago Grijalvo et al.   EXPERT OPINION ON THERAPEUTIC PATENTS

Synthesis and Biophysical Properties of C5-Functionalized LNA (Locked Nucleic Acid)

(2014) Pawan Kumar et al.   JOURNAL OF ORGANIC CHEMISTRY

Liposomal Spherical Nucleic Acids

(2014) Resham J. Banga et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing

(2014) Jayaprakash K. Nair et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

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

Programmable RNA recognition and cleavage by CRISPR/Cas9

(2014) Mitchell R. O’Connell et al.   NATURE

Efficient delivery of RNAi prodrugs containing reversible charge-neutralizing phosphotriester backbone modifications

(2014) Bryan R Meade et al.   NATURE BIOTECHNOLOGY

5′-O-Methylphosphonate nucleic acids—new modified DNAs that increase the Escherichia coli RNase H cleavage rate of hybrid duplexes

(2014) Hana Šípová et al.   NUCLEIC ACIDS RESEARCH

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennaryN-acetyl galactosamine improves potency 10-fold in mice

(2014) Thazha P. Prakash et al.   NUCLEIC ACIDS RESEARCH

Carbohydrate-Functionalized Locked Nucleic Acids: Oligonucleotides with Extraordinary Binding Affinity, Target Specificity, and Enzymatic Stability

(2014) Mamta Kaura et al.   ORGANIC LETTERS

Antisense oligonucleotides: modifications and clinical trials

(2014) Vivek K. Sharma et al.   MedChemComm

10th Annual Meeting of the Oligonucleotide Therapeutics Society

(2014) Keith T. Gagnon et al.   Nucleic Acid Therapeutics

Phosphorothioates, Essential Components of Therapeutic Oligonucleotides

(2014) Fritz Eckstein   Nucleic Acid Therapeutics

Nucleic acid therapeutics: basic concepts and recent developments

(2014) Vivek K. Sharma et al.   RSC Advances

A Kinetic Model Explains Why Shorter and Less Affine Enzyme-recruiting Oligonucleotides Can Be More Potent

(2014) Lykke Pedersen et al.   Molecular Therapy-Nucleic Acids

Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Mipomersen Sodium: First Global Approval

(2013) Philip Hair et al.   DRUGS

Synthesis of Triazole-Linked Oligonucleotides with High Affinity to DNA Complements and an Analysis of Their Compatibility with Biosystems

(2013) Anna M. Varizhuk et al.   JOURNAL OF ORGANIC CHEMISTRY

Mechanism for the endocytosis of spherical nucleic acid nanoparticle conjugates

(2013) C. H. J. Choi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Spherical nucleic acids: A whole new ball game

(2013) S. C. P. Williams   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The prostate-specific membrane antigen: Lessons and current clinical implications from 20 years of research

(2013) Benjamin T. Ristau et al.   UROLOGIC ONCOLOGY-SEMINARS AND ORIGINAL INVESTIGATIONS

Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles

(2013) Barbara L Mui et al.   Molecular Therapy-Nucleic Acids

Application of cationic liposomes for delivery of nucleic acids

(2013) Gayong Shim et al.   Asian Journal of Pharmaceutical Sciences

Polycation-based nanoparticle delivery of RNAi therapeutics: Adverse effects and solutions

(2012) Borja Ballarín-González et al.   ADVANCED DRUG DELIVERY REVIEWS

RNAi-based nanomedicines for targeted personalized therapy

(2012) Ala Daka et al.   ADVANCED DRUG DELIVERY REVIEWS

Single-Stranded siRNAs Activate RNAi in Animals

(2012) Walt F. Lima et al.   CELL

Designing Chemically Modified Oligonucleotides for Targeted Gene Silencing

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

Clinical pharmacokinetics of second generation antisense oligonucleotides

(2012) Rosie Z Yu et al.   Expert Opinion on Drug Metabolism & Toxicology

Small Efficient Cell-penetrating Peptides Derived from Scorpion Toxin Maurocalcine

(2012) Cathy Poillot et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Discovering the first microRNA-targeted drug

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

Antibody-Linked Spherical Nucleic Acids for Cellular Targeting

(2012) Ke Zhang et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

The solution structure of double helical arabino nucleic acids (ANA and 2′F-ANA): effect of arabinoses in duplex-hairpin interconversion

(2012) Nerea Martín-Pintado et al.   NUCLEIC ACIDS RESEARCH

Nonviral delivery of self-amplifying RNA vaccines

(2012) A. J. Geall et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

Discovery and Early Development of AVI-7537 and AVI-7288 for the Treatment of Ebola Virus and Marburg Virus Infections

(2012) Patrick Iversen et al.   Viruses-Basel

Structure Activity Relationships of α-L-LNA Modified Phosphorothioate Gapmer Antisense Oligonucleotides in Animals

(2012) Punit P Seth et al.   Molecular Therapy-Nucleic Acids

Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment

(2012) Corinne Betts et al.   Molecular Therapy-Nucleic Acids

Supramolecular assemblies in functional siRNA delivery: Where do we stand?

(2011) Hamidreza M. Aliabadi et al.   BIOMATERIALS

Synthesis and hybridization data of oligonucleotide analogs with triazole internucleotide linkages, potential antiviral and antitumor agents

(2011) Anna Varizhuk et al.   BIOORGANIC CHEMISTRY

Incorporation of Positively Charged Linkages into DNA and RNA Backbones: A Novel Strategy for Antigene and Antisense Agents

(2011) Moti L. Jain et al.   CHEMICAL REVIEWS

Hybrid lipid oligonucleotide conjugates: synthesis, self-assemblies and biomedical applications

(2011) Amit Patwa et al.   CHEMICAL SOCIETY REVIEWS

An Overview of Sugar-Modified Oligonucleotides for Antisense Therapeutics

(2011) Thazha P. Prakash   CHEMISTRY & BIODIVERSITY

Cellular delivery of siRNA and antisense oligonucleotides via receptor-mediated endocytosis

(2011) Xin Ming   Expert Opinion on Drug Delivery

Chemical Modification of siRNA Bases To Probe and Enhance RNA Interference

(2011) Hayden Peacock et al.   JOURNAL OF ORGANIC CHEMISTRY

Silencing disease genes in the laboratory and the clinic

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

Synthesis, Improved Antisense Activity and Structural Rationale for the Divergent RNA Affinities of 3′-Fluoro Hexitol Nucleic Acid (FHNA and Ara-FHNA) Modified Oligonucleotides

(2011) Martin Egli et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Nanogyroids Incorporating Multivalent Lipids: Enhanced Membrane Charge Density and Pore Forming Ability for Gene Silencing

(2011) Cecília Leal et al.   LANGMUIR

Anti-CD22 Antibody Targeting of pH-responsive Micelles Enhances Small Interfering RNA Delivery and Gene Silencing in Lymphoma Cells

(2011) Maria C Palanca-Wessels et al.   MOLECULAR THERAPY

MicroRNAs 103 and 107 regulate insulin sensitivity

(2011) Mirko Trajkovski et al.   NATURE

Silencing of microRNA families by seed-targeting tiny LNAs

(2011) Susanna Obad et al.   NATURE GENETICS

The long march of antisense

(2011) Dan Jones   NATURE REVIEWS DRUG DISCOVERY

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

Five Years of siRNA Delivery: Spotlight on Gold Nanoparticles

(2011) Abigail K. R. Lytton-Jean et al.   Small

Silencing or Stimulation? siRNA Delivery and the Immune System

(2011) Kathryn A. Whitehead et al.   Annual Review of Chemical and Biomolecular Engineering

Gold Nanoparticles for Biology and Medicine

(2010) David A. Giljohann et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

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

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

Scavenger Receptors Mediate Cellular Uptake of Polyvalent Oligonucleotide-Functionalized Gold Nanoparticles

(2010) Pinal C. Patel et al.   BIOCONJUGATE CHEMISTRY

pH-Responsive Polymeric Micelle Carriers for siRNA Drugs

(2010) A. J. Convertine et al.   BIOMACROMOLECULES

The influence of net charge and charge distribution on cellular uptake and cytosolic localization of arginine-rich peptides

(2010) Likun Fei et al.   JOURNAL OF DRUG TARGETING

Highly Efficient Gene Silencing Activity of siRNA Embedded in a Nanostructured Gyroid Cubic Lipid Matrix

(2010) Cecília Leal et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Polyvalent Oligonucleotide Iron Oxide Nanoparticle “Click” Conjugates

(2010) Joshua I. Cutler et al.   NANO LETTERS

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Targeted Delivery Systems for Oligonucleotide Therapeutics

(2009) Bo Yu et al.   AAPS Journal

Twenty years of cell-penetrating peptides: from molecular mechanisms to therapeutics

(2009) Frederic Heitz et al.   BRITISH JOURNAL OF PHARMACOLOGY

Antisense oligonucleotide pharmacokinetics and metabolism

(2009) Richard S Geary   Expert Opinion on Drug Metabolism & Toxicology

Does inhibition of apolipoprotein B synthesis produce foie gras?

(2009) Kenneth R. Feingold   JOURNAL OF LIPID RESEARCH

Strand Invasion of Extended, Mixed-Sequence B-DNA by γPNAs

(2009) Gaofei He et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Gene Regulation with Polyvalent siRNA−Nanoparticle Conjugates

(2009) David A. Giljohann et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

The Effect of Chemical Modification and Nanoparticle Formulation on Stability and Biodistribution of siRNA in Mice

(2009) Shan Gao et al.   MOLECULAR THERAPY

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

(2009) Aurélie Goyenvalle et al.   MOLECULAR THERAPY

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

(2009) Justin P Dassie et al.   NATURE BIOTECHNOLOGY

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

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

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

siRNA and Innate Immunity

(2009) Marjorie Robbins et al.   OLIGONUCLEOTIDES

Improved Cellular Activity of Antisense Peptide Nucleic Acids by Conjugation to a Cationic Peptide-Lipid (CatLip) Domain

(2008) Uffe Koppelhus et al.   BIOCONJUGATE CHEMISTRY

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

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

Pegaptanib sodium for the treatment of age-related macular degeneration

(2008) Rajendra S Apte   EXPERT OPINION ON PHARMACOTHERAPY

A review of stimuli-responsive nanocarriers for drug and gene delivery

(2008) Srinivas Ganta et al.   JOURNAL OF CONTROLLED RELEASE

Short Antisense Oligonucleotides with Novel 2′−4′ Conformationaly Restricted Nucleoside Analogues Show Improved Potency without Increased Toxicity in Animals

(2008) Punit P. Seth et al.   JOURNAL OF MEDICINAL CHEMISTRY

Fine Tuning of Electrostatics around the Internucleotidic Phosphate through Incorporation of Modified 2′,4′-Carbocyclic-LNAs and -ENAs Leads to Significant Modulation of Antisense Properties

(2008) Chuanzheng Zhou et al.   JOURNAL OF ORGANIC CHEMISTRY

Design, Synthesis, and Properties of 2‘,4‘-BNANC:  A Bridged Nucleic Acid Analogue

(2008) S. M. Abdur Rahman et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Efficient In Vivo Delivery of siRNA to the Liver by Conjugation of α-Tocopherol

(2008) Kazutaka Nishina et al.   MOLECULAR THERAPY

Polyvalent DNA Nanoparticle Conjugates Stabilize Nucleic Acids

(2008) Dwight S. Seferos et al.   NANO LETTERS

Prodrugs: design and clinical applications

(2008) Jarkko Rautio et al.   NATURE REVIEWS DRUG DISCOVERY

OligoWalk: an online siRNA design tool utilizing hybridization thermodynamics

(2008) Z. J. Lu et al.   NUCLEIC ACIDS RESEARCH

Triazole-Linked Analogue of Deoxyribonucleic Acid (TLDNA): Design, Synthesis, and Double-Strand Formation with Natural DNA

(2008) Hiroyuki Isobe et al.   ORGANIC LETTERS

Complications in patients after intravitreal injection of bevacizumab

(2007) Chiharu Shima et al.   ACTA OPHTHALMOLOGICA

Cell penetrating peptide conjugates of steric block oligonucleotides

(2007) Bernard Lebleu et al.   ADVANCED DRUG DELIVERY REVIEWS