Chemical modifications of nucleic acid drugs and their delivery systems for gene-based therapy

Genetic pharmacology: progresses in siRNA delivery and therapeutic applications

(2017) D Scherman et al.   GENE THERAPY

Freezing Directed Construction of Bio/Nano Interfaces: Reagentless Conjugation, Denser Spherical Nucleic Acids, and Better Nanoflares

(2017) Biwu Liu et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Custirsen in combination with docetaxel and prednisone for patients with metastatic castration-resistant prostate cancer (SYNERGY trial): a phase 3, multicentre, open-label, randomised trial

(2017) Kim N Chi et al.   LANCET ONCOLOGY

Immunoprotein-Mediated siRNA Delivery

(2017) Nicole Bäumer et al.   MOLECULAR PHARMACEUTICS

Amphiphilic carbon dots as versatile vectors for nucleic acid and drug delivery

(2017) Hai-Jiao Wang et al.   Nanoscale

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Oligonucleotide therapies for disorders of the nervous system

(2017) Olga Khorkova et al.   NATURE BIOTECHNOLOGY

Cellular uptake and trafficking of antisense oligonucleotides

(2017) Stanley T Crooke et al.   NATURE BIOTECHNOLOGY

Overcoming cellular barriers for RNA therapeutics

(2017) Steven F Dowdy   NATURE BIOTECHNOLOGY

Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

(2017) David R Corey   NATURE NEUROSCIENCE

Intra-endosomal trafficking mediated by lysobisphosphatidic acid contributes to intracellular release of phosphorothioate-modified antisense oligonucleotides

(2017) Shiyu Wang et al.   NUCLEIC ACIDS RESEARCH

Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor

(2017) Karsten Schmidt et al.   NUCLEIC ACIDS RESEARCH

Recent advances in DNAzyme-based gene silencing

(2017) Huanhuan Fan et al.   Science China-Chemistry

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

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

Evaluation of Amphiphilic Peptide Modified Antisense Morpholino Oligonucleotides In Vitro and in Dystrophic mdx Mice

(2017) et al.   Polymers

Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics

(2017) Yuanyu Huang   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

Phase I dose-escalation study of ALN-VSP02, a novel RNAi therapeutic for solid tumors with liver involvement.

(2017) A. Cervantes et al.   JOURNAL OF CLINICAL ONCOLOGY

Technological development of structural DNA/RNA-based RNAi systems and their applications

(2016) Eun Hye Jeong et al.   ADVANCED DRUG DELIVERY REVIEWS

Esterase-Activated Charge-Reversal Polymer for Fibroblast-Exempt Cancer Gene Therapy

(2016) Nasha Qiu et al.   ADVANCED MATERIALS

Vitamin E-Oligo(methyl diglycol l-glutamate) as a Biocompatible and Functional Surfactant for Facile Preparation of Active Tumor-Targeting PLGA Nanoparticles

(2016) Jintian Wu et al.   BIOMACROMOLECULES

Vitamin E-Labeled Polyethylenimine for in vitro and in vivo Gene Delivery

(2016) Jinxing Liu et al.   BIOMACROMOLECULES

Effect of surface properties on liposomal siRNA delivery

(2016) Yuqiong Xia et al.   BIOMATERIALS

Aptamer-integrated DNA nanostructures for biosensing, bioimaging and cancer therapy

(2016) Hong-Min Meng et al.   CHEMICAL SOCIETY REVIEWS

Oligonucleotide conjugates – Candidates for gene silencing therapeutics

(2016) Matt Gooding et al.   EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS

Safety of ASP0113, a cytomegalovirus DNA vaccine, in recipients undergoing allogeneic hematopoietic cell transplantation: an open-label phase 2 trial

(2016) Takehiko Mori et al.   INTERNATIONAL JOURNAL OF HEMATOLOGY

Enhanced gene delivery to the lung using biodegradable polyunsaturated cationic phosphatidylcholine-detergent conjugates

(2016) Philippe Pierrat et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

A phase Ib/IIa study of combination therapy with gemcitabine and Atu027 in patients with locally advanced or metastatic pancreatic adenocarcinoma.

(2016) Beate Schultheis et al.   JOURNAL OF CLINICAL ONCOLOGY

Effect of the REG1 anticoagulation system versus bivalirudin on outcomes after percutaneous coronary intervention (REGULATE-PCI): a randomised clinical trial

(2016) A Michael Lincoff et al.   LANCET

The case for eteplirsen: Paving the way for precision medicine

(2016) M. Carrie Miceli et al.   MOLECULAR GENETICS AND METABOLISM

Aptamers as targeted therapeutics: current potential and challenges

(2016) Jiehua Zhou et al.   NATURE REVIEWS DRUG DISCOVERY

Investigational nanomedicines in 2016: a review of nanotherapeutics currently undergoing clinical trials

(2016) Joseph M. Caster et al.   Wiley Interdisciplinary Reviews-Nanomedicine and Nanobiotechnology

A RNA nanotechnology platform for a simultaneous two-in-one siRNA delivery and its application in synergistic RNAi therapy

(2016) Mihue Jang et al.   Scientific Reports

Photoresponsive Bridged Silsesquioxane Nanoparticles with Tunable Morphology for Light-Triggered Plasmid DNA Delivery

(2015) Yevhen Fatieiev et al.   ACS Applied Materials & Interfaces

Construction of an Aptamer–SiRNA Chimera-Modified Tissue-Engineered Blood Vessel for Cell-Type-Specific Capture and Delivery

(2015) Wen Chen et al.   ACS Nano

Delivery of therapeutic oligonucleotides with cell penetrating peptides

(2015) Prisca Boisguérin et al.   ADVANCED DRUG DELIVERY REVIEWS

Preclinical and clinical development of siRNA-based therapeutics

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

Fusogenic Reactive Oxygen Species Triggered Charge-Reversal Vector for Effective Gene Delivery

(2015) Xin Liu et al.   ADVANCED MATERIALS

Electrostatic Assembly/Disassembly of Nanoscaled Colloidosomes for Light-Triggered Cargo Release

(2015) Song Li et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

A Multi-RNAi Microsponge Platform for Simultaneous Controlled Delivery of Multiple Small Interfering RNAs

(2015) Young Hoon Roh et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Photochemical Regulation of Gene Expression Using Caged siRNAs with Single Terminal Vitamin E Modification

(2015) Yuzhuo Ji et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

DNAzyme-based biosensors and nanodevices

(2015) Liang Gong et al.   CHEMICAL COMMUNICATIONS

DNA enzymes as potential therapeutics: towards clinical application of 10-23 DNAzymes

(2015) Alesya A Fokina et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Cholesterol Modification of p40-Specific Small Interfering RNA Enables Therapeutic Targeting of Dendritic Cells

(2015) Jürgen Brück et al.   JOURNAL OF IMMUNOLOGY

Design and Application of Rolling Circle Amplification for a Tumor-Specific Drug Carrier

(2015) Jong Hwan Kim et al.   JOURNAL OF MEDICINAL CHEMISTRY

First-in-man Phase 1 Clinical Trial of Gene Therapy for Advanced Pancreatic Cancer: Safety, Biodistribution, and Preliminary Clinical Findings

(2015) Louis Buscail et al.   MOLECULAR THERAPY

Clinical experiences with systemically administered siRNA-based therapeutics in cancer

(2015) Jonathan E. Zuckerman et al.   NATURE REVIEWS DRUG DISCOVERY

Design of a platform technology for systemic delivery of siRNA to tumours using rolling circle transcription

(2015) Mihue Jang et al.   Nature Communications

DNA/RNA heteroduplex oligonucleotide for highly efficient gene silencing

(2015) Kazutaka Nishina et al.   Nature Communications

Delivery of cell-penetrating peptide-peptide nucleic acid conjugates by assembly on an oligonucleotide scaffold

(2015) Xing-Liang Zhao et al.   Scientific Reports

Chimeric Antisense Oligonucleotide Conjugated to α-Tocopherol

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

Alicaforsen, an antisense inhibitor of ICAM-1, as treatment for chronic refractory pouchitis after proctocolectomy: A case series

(2015) Thomas Greuter et al.   United European Gastroenterology Journal

Rapid endosomal escape of prickly nanodiamonds: implications for gene delivery

(2015) Zhiqin Chu et al.   Scientific Reports

Self-assembled Messenger RNA Nanoparticles (mRNA-NPs) for Efficient Gene Expression

(2015) Hyejin Kim et al.   Scientific Reports

Effect of pegaptanib sodium 0.3 mg intravitreal injections (Macugen) in intraocular pressure: posthoc analysis from V.I.S.I.O.N. study

(2014) David S Boyer et al.   BRITISH JOURNAL OF OPHTHALMOLOGY

Enzymatic size control of RNA particles using complementary rolling circle transcription (cRCT) method for efficient siRNA production

(2014) Daehoon Han et al.   CHEMICAL COMMUNICATIONS

Multivalent comb-type aptamer–siRNA conjugates for efficient and selective intracellular delivery

(2014) Hyundong Yoo et al.   CHEMICAL COMMUNICATIONS

Living the PCSK9 Adventure: from the Identification of a New Gene in Familial Hypercholesterolemia Towards a Potential New Class of Anticholesterol Drugs

(2014) Marianne Abifadel et al.   Current Atherosclerosis Reports

A phase II trial of intraperitoneal EGEN-001, an IL-12 plasmid formulated with PEG–PEI–cholesterol lipopolymer in the treatment of persistent or recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer: A Gynecologic Oncology Group study

(2014) Ronald D. Alvarez et al.   GYNECOLOGIC ONCOLOGY

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

SNS01-T Modulation of eIF5A Inhibits B-cell Cancer Progression and Synergizes With Bortezomib and Lenalidomide

(2014) Sarah M Francis et al.   MOLECULAR THERAPY

In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight

(2014) James E. Dahlman et al.   Nature Nanotechnology

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

Tumor-targeted in vivo gene silencing via systemic delivery of cRGD-conjugated siRNA

(2014) Xiaoxia Liu 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

Aganirsen Antisense Oligonucleotide Eye Drops Inhibit Keratitis-Induced Corneal Neovascularization and Reduce Need for Transplantation

(2014) Claus Cursiefen et al.   OPHTHALMOLOGY

Correlating animal and human phase Ia/Ib clinical data with CALAA-01, a targeted, polymer-based nanoparticle containing siRNA

(2014) J. E. Zuckerman et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Contracting for Capacity under Renegotiation: Partner Preferences and the Value of Anticipating Renegotiation

(2014) Eda Kemahlıoğlu-Ziya   PRODUCTION AND OPERATIONS MANAGEMENT

Toxicological and Pharmacokinetic Properties of QPI-1007, a Chemically Modified Synthetic siRNA Targeting Caspase 2 mRNA, Following Intravitreal Injection

(2014) Elisabeth C.R. Solano et al.   Nucleic Acid Therapeutics

Functional Nanostructures for Effective Delivery of Small Interfering RNA Therapeutics

(2014) Cheol Am Hong et al.   Theranostics

Silver Nanoscale Antisense Drug Delivery System for Photoactivated Gene Silencing

(2013) Paige K. Brown et al.   ACS Nano

Reducible Hyaluronic Acid–siRNA Conjugate for Target Specific Gene Silencing

(2013) Kitae Park et al.   BIOCONJUGATE CHEMISTRY

Protein-resistant, reductively dissociable polyplexes for in vivo systemic delivery and tumor-targeting of siRNA

(2013) Jee Seon Kim et al.   BIOMATERIALS

The effects of the anti-hepcidin Spiegelmer NOX-H94 on inflammation-induced anemia in cynomolgus monkeys

(2013) F. Schwoebel et al.   BLOOD

The Spiegelmer NOX-A12, a novel CXCL12 inhibitor, interferes with chronic lymphocytic leukemia cell motility and causes chemosensitization

(2013) J. Hoellenriegel et al.   BLOOD

Mipomersen Sodium: First Global Approval

(2013) Philip Hair et al.   DRUGS

Incorporation of histone derived recombinant protein for enhanced disassembly of core-membrane structured liposomal nanoparticles for efficient siRNA delivery

(2013) Yuhua Wang et al.   JOURNAL OF CONTROLLED RELEASE

Cell-penetrating cationic siRNA and lipophilic derivatives efficient at nanomolar concentrations in the presence of serum and albumin

(2013) Phanélie Perche et al.   JOURNAL OF CONTROLLED RELEASE

DNA Based Therapy with Diphtheria Toxin-A BC-819: A Phase 2b Marker Lesion Trial in Patients with Intermediate Risk Nonmuscle Invasive Bladder Cancer

(2013) Ofer N. Gofrit et al.   JOURNAL OF UROLOGY

A Novel C5a-neutralizing Mirror-image (l-)Aptamer Prevents Organ Failure and Improves Survival in Experimental Sepsis

(2013) Kai Hoehlig et al.   MOLECULAR THERAPY

In Vitro and In Vivo Efficacy of SYL040012, a Novel siRNA Compound for Treatment of Glaucoma

(2013) Tamara Martínez et al.   MOLECULAR THERAPY

Delivery materials for siRNA therapeutics

(2013) Rosemary Kanasty et al.   NATURE MATERIALS

Diversifying microRNA sequence and function

(2013) Stefan L. Ameres et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Mutant KRAS is a druggable target for pancreatic cancer

(2013) E. Zorde Khvalevsky et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gene Silencing by Gold Nanoshell-Mediated Delivery and Laser-Triggered Release of Antisense Oligonucleotide and siRNA

(2012) Ryan Huschka et al.   ACS Nano

Targeting von Willebrand factor as a novel anti-platelet therapy; Application of ARC1779, an Anti-vWF aptamer, against thrombotic risk

(2012) Ok-Nam Bae   ARCHIVES OF PHARMACAL RESEARCH

Nano-carrier for gene delivery and bioimaging based on carbon dots with PEI-passivation enhanced fluorescence

(2012) Changjun Liu et al.   BIOMATERIALS

Attacking the genome: emerging siRNA nanocarriers from concept to clinic

(2012) Christopher Alabi et al.   CURRENT OPINION IN PHARMACOLOGY

Oral delivery of small RNA and DNA

(2012) Diane C. Forbes et al.   JOURNAL OF CONTROLLED RELEASE

Light-Triggered Charge Reversal of Organic–Silica Hybrid Nanoparticles

(2012) Li-Chih Hu et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Targeted Delivery of a Splice-Switching Oligonucleotide by Cationic Polyplexes of RGD-Oligonucleotide Conjugate

(2012) Xin Ming et al.   MOLECULAR PHARMACEUTICS

Functional In Vivo Delivery of Multiplexed Anti-HIV-1 siRNAs via a Chemically Synthesized Aptamer With a Sticky Bridge

(2012) Jiehua Zhou et al.   MOLECULAR THERAPY

STING and the innate immune response to nucleic acids in the cytosol

(2012) Dara L Burdette et al.   NATURE IMMUNOLOGY

Self-assembled RNA interference microsponges for efficient siRNA delivery

(2012) Jong Bum Lee et al.   NATURE MATERIALS

Molecularly self-assembled nucleic acid nanoparticles for targeted in vivo siRNA delivery

(2012) Hyukjin Lee et al.   Nature Nanotechnology

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

(2012) Ryszard Kole et al.   NATURE REVIEWS DRUG DISCOVERY

A Highly Entangled Polymeric Nanoconstruct Assembled by siRNA and its Reduction-Triggered siRNA Release for Gene Silencing

(2012) Ran Namgung et al.   Small

Defined Folate-PEG-siRNA Conjugates for Receptor-specific Gene Silencing

(2012) Christian Dohmen et al.   Molecular Therapy-Nucleic Acids

DNA Cage Delivery to Mammalian Cells

(2011) Anthony S. Walsh et al.   ACS Nano

Multivalent Cyclic RGD Conjugates for Targeted Delivery of Small Interfering RNA

(2011) Md. Rowshon Alam et al.   BIOCONJUGATE CHEMISTRY

Current progress of siRNA/shRNA therapeutics in clinical trials

(2011) John C. Burnett et al.   Biotechnology Journal

DermaVir: a plasmid DNA-based nanomedicine therapeutic vaccine for the treatment of HIV/AIDS

(2011) Franco Lori   Expert Review of Vaccines

MicroRNA therapeutics

(2011) J A Broderick et al.   GENE THERAPY

NIR-light triggered delivery of macromolecules into the cytosol

(2011) Susana Carregal-Romero et al.   JOURNAL OF CONTROLLED RELEASE

Light-Induced Release of DNA from Gold Nanoparticles: Nanoshells and Nanorods

(2011) Ryan Huschka et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Efficient In Vivo Delivery of siRNA Into Brain Capillary Endothelial Cells Along With Endogenous Lipoprotein

(2011) Hiroya Kuwahara et al.   MOLECULAR THERAPY

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

SUSTAINED ELEVATED INTRAOCULAR PRESSURES AFTER INTRAVITREAL INJECTION OF BEVACIZUMAB, RANIBIZUMAB, AND PEGAPTANIB

(2011) Daniel Y Choi et al.   RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES

Design, Assembly, and Activity of Antisense DNA Nanostructures

(2011) Jung-Won Keum et al.   Small

Immunogenicity and protective efficacy of a vaxfectin-adjuvanted tetravalent dengue DNA vaccine

(2011) Kevin R. Porter et al.   VACCINE

A synthetic icosahedral DNA-based host–cargo complex for functional in vivo imaging

(2011) Dhiraj Bhatia et al.   Nature Communications

Atu027 Prevents Pulmonary Metastasis in Experimental and Spontaneous Mouse Metastasis Models

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

High-Density Lipoprotein Facilitates In Vivo Delivery of α-Tocopherol–Conjugated Short-Interfering RNA to the Brain

(2010) Yoshitaka Uno et al.   HUMAN GENE THERAPY

Self-assembled siRNA–PLGA conjugate micelles for gene silencing

(2010) Soo Hyeon Lee et al.   JOURNAL OF CONTROLLED RELEASE

Visualizing Light-Triggered Release of Molecules Inside Living Cells

(2010) Ryan Huschka et al.   NANO LETTERS

Aptamers as therapeutics

(2010) Anthony D. Keefe et al.   NATURE REVIEWS DRUG DISCOVERY

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

Cationic siRNAs Provide Carrier-Free Gene Silencing in Animal Cells

(2009) Marc Nothisen et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

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

(2009) Sancy A Leachman et al.   MOLECULAR THERAPY

On the road to reading the RNA-interference code

(2009) Haruhiko Siomi et al.   NATURE

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

siRNA Conjugate Delivery Systems

(2008) Ji Hoon Jeong et al.   BIOCONJUGATE CHEMISTRY

Atu027, a Liposomal Small Interfering RNA Formulation Targeting Protein Kinase N3, Inhibits Cancer Progression

(2008) M. Aleku et al.   CANCER RESEARCH

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

(2008) Priti Kumar et al.   CELL

Neovascular Age-Related Macular Degeneration

(2008) Aimee V Chappelow et al.   DRUGS

The size of endothelial fenestrae in human liver sinusoids: implications for hepatocyte-directed gene transfer

(2008) E Wisse et al.   GENE THERAPY

An efficient and low immunostimulatory nanoparticle formulation for systemic siRNA delivery to the tumor

(2008) Sumio Chono et al.   JOURNAL OF CONTROLLED RELEASE

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

(2008) Natee Jearawiriyapaisarn et al.   MOLECULAR THERAPY

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

(2008) Kazutaka Nishina et al.   MOLECULAR THERAPY

CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression

(2008) Stephen C Hyde et al.   NATURE BIOTECHNOLOGY

Intracellular delivery of an anionic antisense oligonucleotide via receptor-mediated endocytosis

(2008) Md Rowshon Alam et al.   NUCLEIC ACIDS RESEARCH

Development of a Novel Method for Formulating Stable siRNA-Loaded Lipid Particles for In vivo Use

(2008) Sherry Y. Wu et al.   PHARMACEUTICAL RESEARCH

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

(2008) D. Peer et al.   SCIENCE