Delivery of RNA Therapeutics: The Great Endosomal Escape!

Delivery of oligonucleotide‐based therapeutics: challenges and opportunities

(2021) Suzan M Hammond et al.   EMBO Molecular Medicine

Antisense technology: an overview and prospectus

(2021) Stanley T. Crooke et al.   NATURE REVIEWS DRUG DISCOVERY

Chemical Manipulation of the Endosome Trafficking Machinery: Implications for Oligonucleotide Delivery

(2021) Rudolph L. Juliano   Biomedicines

The current landscape of nucleic acid therapeutics

(2021) Jayesh A. Kulkarni et al.   Nature Nanotechnology

Enhanced delivery of peptide-morpholino oligonucleotides with a small molecule to correct splicing defects in the lung

(2021) Yan Dang et al.   NUCLEIC ACIDS RESEARCH

Mechanisms of SARS-CoV-2 entry into cells

(2021) Cody B. Jackson et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Challenges and Opportunities for Nucleic Acid Therapeutics

(2021) David R. Corey et al.   Nucleic Acid Therapeutics

Investigating the pharmacodynamic durability of GalNAc–siRNA conjugates

(2020) Christopher R Brown et al.   NUCLEIC ACIDS RESEARCH

High-resolution visualization and quantification of nucleic acid–based therapeutics in cells and tissues using Nanoscale secondary ion mass spectrometry (NanoSIMS)

(2020) Cuiwen He et al.   NUCLEIC ACIDS RESEARCH

Lipid Conjugates Enhance Endosomal Release of Antisense Oligonucleotides Into Cells

(2019) Shiyu Wang et al.   Nucleic Acid Therapeutics

Enhancing MicroRNA Activity through Increased Endosomal Release Mediated by Nigericin

(2019) Esteban A. Orellana et al.   Molecular Therapy-Nucleic Acids

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

(2019) Julia F. Alterman et al.   NATURE BIOTECHNOLOGY

New Mechanism for Release of Endosomal Contents: Osmotic Lysis via Nigericin-Mediated K+/H+ Exchange

(2018) Loganathan Rangasamy et al.   BIOCONJUGATE CHEMISTRY

Structure–activity relationships and cellular mechanism of action of small molecules that enhance the delivery of oligonucleotides

(2018) Rudolph L Juliano et al.   NUCLEIC ACIDS RESEARCH

Influenza Hemagglutinin Protein Stability, Activation, and Pandemic Risk

(2018) Charles J. Russell et al.   TRENDS IN MICROBIOLOGY

Intracellular Trafficking and Endosomal Release of Oligonucleotides: What We Know and What We Don’t

(2018) R.L. Juliano   Nucleic Acid Therapeutics

RNA Therapeutics (Almost) Comes of Age: Targeting, Delivery and Endosomal Escape

(2018) Steven F. Dowdy et al.   Nucleic Acid Therapeutics

Viral escape from endosomes and host detection at a glance

(2018) Jacqueline Staring et al.   JOURNAL OF CELL SCIENCE

Quantitative fluorescence imaging determines the absolute number of locked nucleic acid oligonucleotides needed for suppression of target gene expression

(2018) Annette Buntz et al.   NUCLEIC ACIDS RESEARCH

Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo

(2018) Annabelle Biscans et al.   NUCLEIC ACIDS RESEARCH

Saponins enhance exon skipping of 2′-O-methyl phosphorothioate oligonucleotide in vitro and in vivo

(2018) Mingxing Wang et al.   Drug Design Development and Therapy

Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment

(2018) Michael J. Gait et al.   Nucleic Acid Therapeutics

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Overcoming cellular barriers for RNA therapeutics

(2017) Steven F Dowdy   NATURE BIOTECHNOLOGY

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9

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

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

Protease-triggered siRNA delivery vehicles

(2015) David B. Rozema et al.   JOURNAL OF CONTROLLED RELEASE

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

(2015) Anders Wittrup et al.   NATURE BIOTECHNOLOGY

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

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

Improving the In Vivo Therapeutic Index of siRNA Polymer Conjugates through Increasing pH Responsiveness

(2014) Erin N. Guidry et al.   BIOCONJUGATE CHEMISTRY

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

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

(2014) Bryan R Meade et al.   NATURE BIOTECHNOLOGY

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

Image-based analysis of lipid nanoparticle–mediated siRNA delivery, intracellular trafficking and endosomal escape

(2013) Jerome Gilleron et al.   NATURE BIOTECHNOLOGY

The small molecule Retro-1 enhances the pharmacological actions of antisense and splice switching oligonucleotides

(2013) Xin Ming et al.   NUCLEIC ACIDS RESEARCH

Modeling the Step of Endosomal Escape during Cell Infection by a Nonenveloped Virus

(2012) Thibault Lagache et al.   BIOPHYSICAL JOURNAL

Glucan particles for selective delivery of siRNA to phagocytic cells in mice

(2011) Gregory J. Tesz et al.   BIOCHEMICAL JOURNAL

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY