Non-viral vectors for gene-based therapy

Multiplex Genome Engineering Using CRISPR/Cas Systems

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RNA-Guided Human Genome Engineering via Cas9

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A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine.

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Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure.

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

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Factors affecting the clearance and biodistribution of polymeric nanoparticles

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One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering

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The druggable genome

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Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity

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Knocking down barriers: advances in siRNA delivery

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Biomolecular coronas provide the biological identity of nanosized materials

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Suppression of RNA Recognition by Toll-like Receptors: The Impact of Nucleoside Modification and the Evolutionary Origin of RNA

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

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A microRNA component of the p53 tumour suppressor network

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Design and development of polymers for gene delivery

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

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Progress and problems with the use of viral vectors for gene therapy

Thomas, C. E., Ehrhardt, A., & Kay, M. A. (2003) · Nature Reviews Genetics · doi:10.1038/nrg1066 · Cited by 2,501 · see citing works

Nonviral vectors for gene delivery

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Asymmetry in the Assembly of the RNAi Enzyme Complex

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Toxicity of cationic lipids and cationic polymers in gene delivery

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Genome editing with engineered zinc finger nucleases

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A TALE nuclease architecture for efficient genome editing

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Designing dendrimers for biological applications

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A Simple Cipher Governs DNA Recognition by TAL Effectors

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

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Rational design of cationic lipids for siRNA delivery

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Delivery materials for siRNA therapeutics

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MicroRNA profiling: approaches and considerations

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Phenotypic Heterogeneity of the Endothelium

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Nanoparticle Delivery of Cancer Drugs

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The evolution of gene regulation by transcription factors and microRNAs

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RNAi-mediated gene silencing in non-human primates

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An Inverted Hexagonal Phase of Cationic Liposome-DNA Complexes Related to DNA Release and Delivery

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Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo

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Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms

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Phenotypic Heterogeneity of the Endothelium

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Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs

Morrissey, D. V., Lockridge, J. A., Shaw, L., Blanchard, K. S., Jensen, K., Breen, W., Hartsough, K. C., Machemer, L., Radka, S. F., Jadhav, V., et al. (2005) · Nature Biotechnology · doi:10.1038/nbt1122 · Cited by 1,168 · see citing works

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

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

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High efficiency transformation by direct microinjection of DNA into cultured mammalian cells

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

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Polyethylenimine-based non-viral gene delivery systems

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

Davis, M. E. (2009) · Molecular Pharmaceutics · doi:10.1021/mp900015y · Cited by 979 · see citing works

Mechanisms and optimization of in vivo delivery of lipophilic siRNAs

Wolfrum, C., Shi, S., Jayaprakash, K. N., Jayaraman, M., Wang, G., Pandey, R. K., Rajeev, K. G., Nakayama, T., Charrise, K., Ndungo, E. M., et al. (2007) · Nature Biotechnology · doi:10.1038/nbt1339 · Cited by 974 · see citing works

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

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

Yin, H., Xue, W., Chen, S., Bogorad, R. L., Benedetti, E., Grompe, M., Koteliansky, V., Sharp, P. A., Jacks, T., & Anderson, D. G. (2014) · Nature Biotechnology · doi:10.1038/nbt.2884 · Cited by 951 · see citing works

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

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

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Receptor-mediated in vitro gene transformation by a soluble DNA carrier system.

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Polymersomes

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Tracking the intracellular path of poly(ethylenimine)/DNA complexes for gene delivery

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Gene therapy clinical trials worldwide to 2012 – an update

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

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PEGylation significantly affects cellular uptake and intracellular trafficking of non-viral gene delivery particles

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

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

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Different behavior of branched and linear polyethylenimine for gene deliveryin vitro andin vivo

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Engineering targeted viral vectors for gene therapy

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Therapeutic EphA2 Gene Targeting In vivo Using Neutral Liposomal Small Interfering RNA Delivery

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

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

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Dynamic PolyConjugates for targeted in vivo delivery of siRNA to hepatocytes

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Design and gene delivery activity of modified polyethylenimines

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

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Lipid-based Nanoparticles for Nucleic Acid Delivery

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Immune responses to gene therapy vectors: influence on vector function and effector mechanisms

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Receptor-mediated gene delivery and expression in vivo.

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Stealth Me. PEG‐PLA Nanoparticles Avoid Uptake by the Mononuclear Phagocytes System

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In vivo activity of nuclease-resistant siRNAs

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Inorganic Nanoparticles as Carriers of Nucleic Acids into Cells

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Cationic lipids, lipoplexes and intracellular delivery of genes.

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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

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Polymers for gene delivery across length scales

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Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids

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Sequence-Specific Knockdown of EWS-FLI1 by Targeted, Nonviral Delivery of Small Interfering RNA Inhibits Tumor Growth in a Murine Model of Metastatic Ewing's Sarcoma

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

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Polyethylenimine-graft-Poly(ethylene glycol) Copolymers: Influence of Copolymer Block Structure on DNA Complexation and Biological Activities as Gene Delivery System

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Vector unpacking as a potential barrier for receptor-mediated polyplex gene delivery

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Enhancing polyethylenimine's delivery of plasmid DNA into mammalian cells

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Non-viral gene therapy: polycation-mediated DNA delivery

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Barriers to Nonviral Gene Delivery

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

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The Fate of Plasmid DNA After Intravenous Injection in Mice: Involvement of Scavenger Receptors in Its Hepatic Uptake

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Current progress of siRNA/shRNA therapeutics in clinical trials

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Breaking up the correlation between efficacy and toxicity for nonviral gene delivery

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Introduction of liposome-encapsulated SV40 DNA into cells.

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Physical methods for gene transfer: Improving the kinetics of gene delivery into cells

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Gene therapy progress and prospects: Ultrasound for gene transfer

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Administration in non-human primates of escalating intravenous doses of targeted nanoparticles containing ribonucleotide reductase subunit M2 siRNA

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

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Polyethylenimine-based intravenous delivery of transgenes to mouse lung

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Gene Therapy Progress and Prospects: Electroporation and other physical methods

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The Magnetofection Method: Using Magnetic Force to Enhance Gene Delivery

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A novel siRNA-lipoplex technology for RNA interference in the mouse vascular endothelium

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Mutagenesis and Oncogenesis by Chromosomal Insertion of Gene Transfer Vectors

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