Non-viral vectors for gene-based therapy

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering

(2013) Haoyi Wang et al.   CELL

Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity

(2013) F. Ann Ran et al.   CELL

Transfection efficiency and transgene expression kinetics of mRNA delivered in naked and nanoparticle format

(2013) Kyle K.L. Phua et al.   JOURNAL OF CONTROLLED RELEASE

Gene therapy clinical trials worldwide to 2012 - an update

(2013) Samantha L. Ginn et al.   JOURNAL OF GENE MEDICINE

Intralesional immunotherapy for melanoma

(2013) Peter Hersey et al.   JOURNAL OF SURGICAL ONCOLOGY

Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial

(2013) Kevin Fitzgerald et al.   LANCET

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

(2013) Christine I Wooddell et al.   MOLECULAR THERAPY

Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction

(2013) Lior Zangi et al.   NATURE BIOTECHNOLOGY

Efficiency of siRNA delivery by lipid nanoparticles is limited by endocytic recycling

(2013) Gaurav Sahay et al.   NATURE BIOTECHNOLOGY

Delivery materials for siRNA therapeutics

(2013) Rosemary Kanasty et al.   NATURE MATERIALS

Transferrin-functionalized nanoparticles lose their targeting capabilities when a biomolecule corona adsorbs on the surface

(2013) Anna Salvati et al.   Nature Nanotechnology

Multiparametric approach for the evaluation of lipid nanoparticles for siRNA delivery

(2013) C. A. Alabi 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

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

(2013) Thomas Gaj et al.   TRENDS IN BIOTECHNOLOGY

A CRISPR way to engineer the human genome

(2013) Sivaprakash Ramalingam et al.   GENOME BIOLOGY

Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo

(2012) Muthusamy Jayaraman et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

In vivo disassembly of IV administered siRNA matrix nanoparticles at the renal filtration barrier

(2012) Broes Naeye et al.   BIOMATERIALS

Clinical Applications for microRNAs in Cancer

(2012) S P Nana-Sinkam et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Attacking the genome: emerging siRNA nanocarriers from concept to clinic

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

Action and Reaction: The Biological Response to siRNA and Its Delivery Vehicles

(2012) Rosemary L Kanasty et al.   MOLECULAR THERAPY

Increased Erythropoiesis in Mice Injected With Submicrogram Quantities of Pseudouridine-containing mRNA Encoding Erythropoietin

(2012) Katalin Karikó et al.   MOLECULAR THERAPY

Biomolecular coronas provide the biological identity of nanosized materials

(2012) Marco P. Monopoli et al.   Nature Nanotechnology

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

(2012) Hyukjin Lee et al.   Nature Nanotechnology

MicroRNA profiling: approaches and considerations

(2012) Colin C. Pritchard et al.   NATURE REVIEWS GENETICS

Polycation-siRNA nanoparticles can disassemble at the kidney glomerular basement membrane

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

Lipid-derived nanoparticles for immunostimulatory RNA adjuvant delivery

(2012) D. N. Nguyen et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

miR-34 – a microRNA replacement therapy is headed to the clinic

(2012) Andreas G. Bader   Frontiers in Genetics

Nanoparticle Delivery of Cancer Drugs

(2011) Andrew Z. Wang et al.   Annual Review of Medicine

Current progress of siRNA/shRNA therapeutics in clinical trials

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

The Sleeping Beauty transposon system: a non-viral vector for gene therapy

(2011) E. L. Aronovich et al.   HUMAN MOLECULAR GENETICS

In Vitroandin VivomRNA Delivery Using Lipid-Enveloped pH-Responsive Polymer Nanoparticles

(2011) Xingfang Su et al.   MOLECULAR PHARMACEUTICS

Systemic Delivery of Tumor Suppressor microRNA Mimics Using a Neutral Lipid Emulsion Inhibits Lung Tumors in Mice

(2011) Phong Trang et al.   MOLECULAR THERAPY

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

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

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

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

State-of-the-art gene-based therapies: the road ahead

(2011) Mark A. Kay   NATURE REVIEWS GENETICS

Nucleoside modifications in RNA limit activation of 2'-5'-oligoadenylate synthetase and increase resistance to cleavage by RNase L

(2011) B. R. Anderson et al.   NUCLEIC ACIDS RESEARCH

Silencing or Stimulation? siRNA Delivery and the Immune System

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

Tumor-Selective Delivery of Macromolecular Drugs via the EPR Effect: Background and Future Prospects

(2010) Hiroshi Maeda   BIOCONJUGATE CHEMISTRY

Postexposure protection of non-human primates against a lethal Ebola virus challenge with RNA interference: a proof-of-concept study

(2010) Thomas W Geisbert et al.   LANCET

Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms

(2010) Akin Akinc et al.   MOLECULAR THERAPY

Elimination Pathways of Systemically Delivered siRNA

(2010) Yuanyu Huang et al.   MOLECULAR THERAPY

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

(2010) Mark E. Davis et al.   NATURE

A robust system for production of minicircle DNA vectors

(2010) Mark A Kay et al.   NATURE BIOTECHNOLOGY

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

Genome editing with engineered zinc finger nucleases

(2010) Fyodor D. Urnov et al.   NATURE REVIEWS GENETICS

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

Targeted Delivery Systems for Oligonucleotide Therapeutics

(2009) Bo Yu et al.   AAPS Journal

Tissue-specific and transcription factor-mediated nuclear entry of DNA

(2009) Aaron M. Miller et al.   ADVANCED DRUG DELIVERY REVIEWS

Progress and Prospects: The design and production of plasmid vectors

(2009) D R Gill et al.   GENE THERAPY

Quantification of plasmid DNA copies in the nucleus after lipoplex and polyplex transfection

(2009) Richard N. Cohen et al.   JOURNAL OF CONTROLLED RELEASE

The First Targeted Delivery of siRNA in Humans via a Self-Assembling, Cyclodextrin Polymer-Based Nanoparticle: From Concept to Clinic

(2009) Mark E. Davis   MOLECULAR PHARMACEUTICS

Development of Lipidoid–siRNA Formulations for Systemic Delivery to the Liver

(2009) Akin Akinc et al.   MOLECULAR THERAPY

Knocking down barriers: advances in siRNA delivery

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

Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application

(2009) Aimee L. Jackson et al.   NATURE REVIEWS DRUG DISCOVERY

Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors

(2009) J. Boch et al.   SCIENCE

A Simple Cipher Governs DNA Recognition by TAL Effectors

(2009) M. J. Moscou et al.   SCIENCE

In vivo pharmacokinetics, tissue distribution and underlying mechanisms of various PEI(–PEG)/siRNA complexes

(2009) Anastasia Malek et al.   TOXICOLOGY AND APPLIED PHARMACOLOGY

Progress in developing cationic vectors for non-viral systemic gene therapy against cancer

(2008) Marie Morille et al.   BIOMATERIALS

Viral vectors: from virology to transgene expression

(2008) D Bouard et al.   BRITISH JOURNAL OF PHARMACOLOGY

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

(2008) M. Aleku et al.   CANCER RESEARCH

Nonviral Vectors for Gene Delivery

(2008) Meredith A. Mintzer et al.   CHEMICAL REVIEWS

Persistent episomal transgene expression in liver following delivery of a scaffold/matrix attachment region containing non-viral vector

(2008) O Argyros et al.   GENE THERAPY

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

(2008) E Wisse et al.   GENE THERAPY

Sustained liver-specific transgene expression from the albumin promoter in mice following hydrodynamic plasmid DNA delivery

(2008) Christine I. Wooddell et al.   JOURNAL OF GENE MEDICINE

Factors Affecting the Clearance and Biodistribution of Polymeric Nanoparticles

(2008) Frank Alexis et al.   MOLECULAR PHARMACEUTICS

Cationic liposomal lipids: From gene carriers to cell signaling

(2008) Caroline Lonez et al.   PROGRESS IN LIPID RESEARCH

Inorganic Nanoparticles as Carriers of Nucleic Acids into Cells

(2007) Viktoriya Sokolova et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Impact of tumor-specific targeting and dosing schedule on tumor growth inhibition after intravenous administration of siRNA-containing nanoparticles

(2007) Derek W. Bartlett et al.   BIOTECHNOLOGY AND BIOENGINEERING