Overcoming cellular barriers for RNA therapeutics

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9

(2017) Kevin Fitzgerald et al.   NEW ENGLAND JOURNAL OF MEDICINE

Cryo-electron microscopy for structure analyses of membrane proteins in the lipid bilayer

(2016) Kazuhiro Abe et al.   CURRENT OPINION IN STRUCTURAL BIOLOGY

Intrathecal Injections in Children With Spinal Muscular Atrophy

(2016) Manon Haché et al.   JOURNAL OF CHILD NEUROLOGY

A Retrospective 30 Years After Discovery of the Enhanced Permeability and Retention Effect of Solid Tumors: Next-Generation Chemotherapeutics and Photodynamic Therapy-Problems, Solutions, and Prospects

(2016) Hiroshi Maeda et al.   MICROCIRCULATION

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy

(2016) Claudia A. Chiriboga et al.   NEUROLOGY

Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs

(2016) Jie Jiang et al.   NEURON

Hsp90 protein interacts with phosphorothioate oligonucleotides containing hydrophobic 2′-modifications and enhances antisense activity

(2016) Xue-Hai Liang et al.   NUCLEIC ACIDS RESEARCH

The delivery of therapeutic oligonucleotides

(2016) Rudolph L. Juliano   NUCLEIC ACIDS RESEARCH

Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles

(2016) Ming Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Disposition and Pharmacology of a GalNAc3-conjugated ASO Targeting Human Lipoprotein (a) in Mice

(2016) Rosie Z Yu et al.   Molecular Therapy-Nucleic Acids

Enhancing Endosomal Escape for Intracellular Delivery of Macromolecular Biologic Therapeutics

(2016) Peter Lönn et al.   Scientific Reports

A role for peptides in overcoming endosomal entrapment in siRNA delivery — A focus on melittin

(2015) Kirk K. Hou et al.   BIOTECHNOLOGY ADVANCES

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

Cationic PTD/CPP-mediated macromolecular delivery: charging into the cell

(2015) Peter Lönn et al.   Expert Opinion on Drug Delivery

Gene-targeting pharmaceuticals for single-gene disorders

(2015) Arthur L. Beaudet et al.   HUMAN MOLECULAR GENETICS

Discovery and Characterization of a Peptide That Enhances Endosomal Escape of Delivered Proteins in Vitro and in Vivo

(2015) Margie Li et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Don’t edit the human germ line

(2015) Edward Lanphier et al.   NATURE

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Visualizing lipid-formulated siRNA release from endosomes and target gene knockdown

(2015) Anders Wittrup et al.   NATURE BIOTECHNOLOGY

An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia

(2015) Alfica Sehgal et al.   NATURE MEDICINE

Efficient delivery of nuclease proteins for genome editing in human stem cells and primary cells

(2015) Jia Liu et al.   Nature Protocols

From guide to target: molecular insights into eukaryotic RNA-interference machinery

(2015) Jonathan J Ipsaro et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Guanabenz (Wytensin™) selectively enhances uptake and efficacy of hydrophobically modified siRNAs

(2015) Maire F. Osborn et al.   NUCLEIC ACIDS RESEARCH

High-throughput screening identifies small molecules that enhance the pharmacological effects of oligonucleotides

(2015) B. Yang et al.   NUCLEIC ACIDS RESEARCH

A cytoplasmic pathway for gapmer antisense oligonucleotide-mediated gene silencing in mammalian cells

(2015) Daniela Castanotto et al.   NUCLEIC ACIDS RESEARCH

Identification and characterization of intracellular proteins that bind oligonucleotides with phosphorothioate linkages

(2015) Xue-hai Liang et al.   NUCLEIC ACIDS RESEARCH

A prudent path forward for genomic engineering and germline gene modification

(2015) D. Baltimore et al.   SCIENCE

From guide to target: molecular insights into eukaryotic RNA-interference machinery

(2015) Jonathan J Ipsaro et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Progress Toward Synthetic Cells

(2014) J. Craig Blain et al.   Annual Review of Biochemistry

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

A Cationic Nanoemulsion for the Delivery of Next-generation RNA Vaccines

(2014) Luis A Brito et al.   MOLECULAR THERAPY

DNA interrogation by the CRISPR RNA-guided endonuclease Cas9

(2014) Samuel H. Sternberg et al.   NATURE

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

(2014) Bryan R Meade et al.   NATURE BIOTECHNOLOGY

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo

(2014) John A Zuris et al.   NATURE BIOTECHNOLOGY

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Synthesis, biophysical properties and biological activity of second generation antisense oligonucleotides containing chiral phosphorothioate linkages

(2014) W. Brad Wan et al.   NUCLEIC ACIDS RESEARCH

Systematic evaluation of antibody-mediated siRNA delivery using an industrial platform of THIOMAB–siRNA conjugates

(2014) Trinna L. Cuellar 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

Cellular Uptake and Intracellular Trafficking of Oligonucleotides: Implications for Oligonucleotide Pharmacology

(2014) R.L. Juliano et al.   Nucleic Acid Therapeutics

Phosphorothioates, Essential Components of Therapeutic Oligonucleotides

(2014) Fritz Eckstein   Nucleic Acid Therapeutics

The “Strong” RNA World Hypothesis: Fifty Years Old

(2013) Marc Neveu et al.   ASTROBIOLOGY

Efficient Generation of Human iPSCs by a Synthetic Self-Replicative RNA

(2013) Naohisa Yoshioka et al.   Cell Stem Cell

Influence of diverse chemical modifications on the ADME characteristics and toxicology of antisense oligonucleotides

(2013) Mehrdad Dirin et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Novel Hydrophobically Modified Asymmetric RNAi Compounds (sd-rxRNA) Demonstrate Robust Efficacy in the Eye

(2013) Michael Byrne et al.   JOURNAL OF OCULAR PHARMACOLOGY AND THERAPEUTICS

RNA-Based Vaccination: Sending a Strong Message

(2013) David B. Weiner   MOLECULAR THERAPY

Hepatocyte-targeted RNAi Therapeutics for the Treatment of Chronic Hepatitis B Virus Infection

(2013) Christine I Wooddell et al.   MOLECULAR THERAPY

Delivery materials for siRNA therapeutics

(2013) Rosemary Kanasty et al.   NATURE MATERIALS

Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration

(2013) C. Lagier-Tourenne et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

A Chemical View of Oligonucleotides for Exon Skipping and Related Drug Applications

(2013) Peter Järver et al.   Nucleic Acid Therapeutics

Optimized siRNA-PEG Conjugates for Extended Blood Circulation and Reduced Urine Excretion in Mice

(2013) Frank Iversen et al.   Theranostics

Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression

(2012) Dongbo Yu et al.   CELL

Intracellular Localization and Routing of miRNA and RNAi Pathway Components

(2012) Thomas Ohrt et al.   CURRENT TOPICS IN MEDICINAL CHEMISTRY

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

(2012) Holly B. Kordasiewicz et al.   NEURON

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

(2012) M. Jinek et al.   SCIENCE

Progress Toward In Vivo Use of siRNAs-II

(2011) Garrett R Rettig et al.   MOLECULAR THERAPY

Expression of therapeutic proteins after delivery of chemically modified mRNA in mice

(2011) Michael S D Kormann et al.   NATURE BIOTECHNOLOGY

Long pre-mRNA depletion and RNA missplicing contribute to neuronal vulnerability from loss of TDP-43

(2011) Magdalini Polymenidou et al.   NATURE NEUROSCIENCE

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

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

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

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

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

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

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Lipid-like materials for low-dose, in vivo gene silencing

(2010) K. T. Love et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Mechanisms of Endocytosis

(2009) Gary J. Doherty et al.   Annual Review of Biochemistry

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

(2009) Chan Il Chang et al.   MOLECULAR THERAPY

Nucleation, propagation and cleavage of target RNAs in Ago silencing complexes

(2009) Yanli Wang et al.   NATURE

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

Solid-Phase Synthesis of Stereoregular Oligodeoxyribonucleoside Phosphorothioates Using Bicyclic Oxazaphospholidine Derivatives as Monomer Units

(2008) Natsuhisa Oka et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Incorporation of Pseudouridine Into mRNA Yields Superior Nonimmunogenic Vector With Increased Translational Capacity and Biological Stability

(2008) Katalin Karikó et al.   MOLECULAR THERAPY

Asymmetric RNA duplexes mediate RNA interference in mammalian cells

(2008) Xiangao Sun et al.   NATURE BIOTECHNOLOGY

A combinatorial library of lipid-like materials for delivery of RNAi therapeutics

(2008) Akin Akinc et al.   NATURE BIOTECHNOLOGY