Non-viral vectors for gene-based therapy (Q34429032)

2 August 2017

title

Non-viral vectors for gene-based therapy (English)

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2 August 2017

Hao Yin

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2 August 2017

Rosemary L Kanasty

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2 August 2017

Ahmed A Eltoukhy

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2 August 2017

Arturo J Vegas

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2 August 2017

J Robert Dorkin

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2 August 2017

Daniel G Anderson

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2 August 2017

publication date

15 July 2014

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2 August 2017

published in

Nature Reviews Genetics

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2 August 2017

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2 August 2017

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2 August 2017

page(s)

541-555

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Gene therapy clinical trials worldwide to 2012 - an update.

2 August 2017

cites work

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

31 March 2020

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

31 March 2020

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Mutagenesis and oncogenesis by chromosomal insertion of gene transfer vectors.

31 March 2020

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

31 March 2020

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Engineering targeted viral vectors for gene therapy

31 March 2020

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

31 March 2020

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Viral vectors: from virology to transgene expression

31 March 2020

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Design and development of polymers for gene delivery

31 March 2020

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Nonviral vectors for gene delivery

31 March 2020

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Polymers for gene delivery across length scales.

31 March 2020

1 reference

The first targeted delivery of siRNA in humans via a self-assembling, cyclodextrin polymer-based nanoparticle: from concept to clinic.

31 March 2020

1 reference

New class of polymers for the delivery of macromolecular therapeutics

31 March 2020

1 reference

Rational design of cationic lipids for siRNA delivery

31 March 2020

1 reference

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

31 March 2020

1 reference

Biomolecular coronas provide the biological identity of nanosized materials

31 March 2020

1 reference

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

31 March 2020

1 reference

Suppression of RNA recognition by Toll-like receptors: the impact of nucleoside modification and the evolutionary origin of RNA

31 March 2020

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

31 March 2020

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Increased erythropoiesis in mice injected with submicrogram quantities of pseudouridine-containing mRNA encoding erythropoietin

31 March 2020

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Chemical modification of siRNA.

31 March 2020

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

31 March 2020

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Gene therapy progress and prospects: electroporation and other physical methods.

31 March 2020

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Gene therapy progress and prospects: ultrasound for gene transfer

31 March 2020

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The magnetofection method: using magnetic force to enhance gene delivery.

31 March 2020

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Lipid-based nanoparticles for nucleic acid delivery

31 March 2020

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Non-viral gene therapy: polycation-mediated DNA delivery

31 March 2020

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Designing dendrimers for biological applications

31 March 2020

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31 March 2020

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Peptide-guided gene delivery

31 March 2020

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Inorganic nanoparticles as carriers of nucleic acids into cells

31 March 2020

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The fate of plasmid DNA after intravenous injection in mice: involvement of scavenger receptors in its hepatic uptake

31 March 2020

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Observation of a Rectangular Columnar Phase in a DNA−Calcium−Zwitterionic Lipid Complex

31 March 2020

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An inverted hexagonal phase of cationic liposome-DNA complexes related to DNA release and delivery

31 March 2020

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Barriers to nonviral gene delivery

31 March 2020

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

31 March 2020

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

31 March 2020

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

31 March 2020

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Quantitative comparison of polyethylenimine formulations and adenoviral vectors in terms of intracellular gene delivery processes

31 March 2020

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Understanding intracellular transport processes pertinent to synthetic gene delivery via stochastic simulations and sensitivity analyses

31 March 2020

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Cationic lipids, lipoplexes and intracellular delivery of genes.

31 March 2020

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

31 March 2020

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Gene delivery with low molecular weight linear polyethylenimines.

31 March 2020

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

31 March 2020

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

31 March 2020

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Progress and prospects: the design and production of plasmid vectors.

31 March 2020

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Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor 1alpha promoter.

31 March 2020

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

31 March 2020

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Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro

31 March 2020

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

31 March 2020

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Plasmid DNA size does not affect the physicochemical properties of lipoplexes but modulates gene transfer efficiency

31 March 2020

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Minicircle: an improved DNA molecule for in vitro and in vivo gene transfer

31 March 2020

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A robust system for production of minicircle DNA vectors

31 March 2020

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A direct comparison of two nonviral gene therapy vectors for somatic integration: in vivo evaluation of the bacteriophage integrase phiC31 and the Sleeping Beauty transposase

31 March 2020

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piggyBac is a flexible and highly active transposon as compared to sleeping beauty, Tol2, and Mos1 in mammalian cells

31 March 2020

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

31 March 2020

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

31 March 2020

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Knocking down barriers: advances in siRNA delivery

31 March 2020

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Cationic liposomal lipids: from gene carriers to cell signaling

31 March 2020

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Intralesional immunotherapy for melanoma

31 March 2020

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Model nucleoprotein complexes: Studies on the interaction of cationic homopolypeptides with DNA

31 March 2020

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Characterization of DNA condensates induced by poly(ethylene oxide) and polylysine

31 March 2020

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Polyethylene glycol-grafted poly-L-lysine as polymeric gene carrier

31 March 2020

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Factors affecting the clearance and biodistribution of polymeric nanoparticles.

31 March 2020

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

31 March 2020

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Compacted DNA nanoparticles administered to the nasal mucosa of cystic fibrosis subjects are safe and demonstrate partial to complete cystic fibrosis transmembrane regulator reconstitution

31 March 2020

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Polyethylenimine-based non-viral gene delivery systems

31 March 2020

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

31 March 2020

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Size matters: molecular weight affects the efficiency of poly(ethylenimine) as a gene delivery vehicle

31 March 2020

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Different behavior of branched and linear polyethylenimine for gene delivery in vitro and in vivo

31 March 2020

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Polyethylenimine-based intravenous delivery of transgenes to mouse lung.

31 March 2020

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Design and gene delivery activity of modified polyethylenimines.

31 March 2020

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In vivo delivery to tumors of DNA complexed with linear polyethylenimine.

31 March 2020

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Toxicity of cationic lipids and cationic polymers in gene delivery

31 March 2020

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Evaluation of polyether-polyethyleneimine graft copolymers as gene transfer agents.

31 March 2020

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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

31 March 2020

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

31 March 2020

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Enhancing polyethylenimine's delivery of plasmid DNA into mammalian cells

31 March 2020

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Highly Effective Gene Transfection In Vivo by Alkylated Polyethylenimine

31 March 2020

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

31 March 2020

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

31 March 2020

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In Vitro and in Vivo mRNA Delivery Using Lipid-Enveloped pH-Responsive Polymer Nanoparticles

31 March 2020

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

31 March 2020

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Action and reaction: the biological response to siRNA and its delivery vehicles

31 March 2020

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In vivo activity of nuclease-resistant siRNAs

31 March 2020

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

31 March 2020

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Lipid-derived nanoparticles for immunostimulatory RNA adjuvant delivery

31 March 2020

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Silencing or stimulation? siRNA delivery and the immune system

31 March 2020

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Nanoparticle delivery of cancer drugs.

31 March 2020

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Mechanisms and optimization of in vivo delivery of lipophilic siRNAs.

31 March 2020

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

31 March 2020

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

31 March 2020

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Elimination Pathways of Systemically Delivered siRNA

31 March 2020

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Dynamic PolyConjugates for targeted in vivo delivery of siRNA to hepatocytes

31 March 2020

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Delivery materials for siRNA therapeutics

31 March 2020

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

31 March 2020

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

31 March 2020

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Phenotypic heterogeneity of the endothelium: I. Structure, function, and mechanisms

31 March 2020

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

31 March 2020

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Tumor-selective delivery of macromolecular drugs via the EPR effect: background and future prospects

31 March 2020

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Targeted delivery systems for oligonucleotide therapeutics

31 March 2020

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

31 March 2020

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

31 March 2020

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Asymmetry in the assembly of the RNAi enzyme complex

31 March 2020

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

31 March 2020

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RNAi-mediated gene silencing in non-human primates.

31 March 2020

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Stabilized plasmid-lipid particles: construction and characterization

31 March 2020

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

31 March 2020

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

31 March 2020

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Development of lipidoid-siRNA formulations for systemic delivery to the liver

31 March 2020

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Multiparametric approach for the evaluation of lipid nanoparticles for siRNA delivery

31 March 2020

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Current progress of siRNA/shRNA therapeutics in clinical trials

31 March 2020

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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

31 March 2020

1 reference

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

31 March 2020

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RNA interference in the mouse vascular endothelium by systemic administration of siRNA-lipoplexes for cancer therapy

31 March 2020

1 reference

Atu027, a liposomal small interfering RNA formulation targeting protein kinase N3, inhibits cancer progression.

31 March 2020

1 reference

Therapeutic EphA2 gene targeting in vivo using neutral liposomal small interfering RNA delivery

31 March 2020

1 reference

Self-assembling nucleic acid delivery vehicles via linear, water-soluble, cyclodextrin-containing polymers

31 March 2020

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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

31 March 2020

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

31 March 2020

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

31 March 2020

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

31 March 2020

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Co-injection of a targeted, reversibly masked endosomolytic polymer dramatically improves the efficacy of cholesterol-conjugated small interfering RNAs in vivo

31 March 2020

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Endosomolysis by masking of a membrane-active agent (EMMA) for cytoplasmic release of macromolecules

31 March 2020

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Hepatocyte-targeted RNAi therapeutics for the treatment of chronic hepatitis B virus infection

31 March 2020

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RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice

31 March 2020

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The evolution of gene regulation by transcription factors and microRNAs

31 March 2020

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MicroRNA profiling: approaches and considerations

31 March 2020

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Clinical applications for microRNAs in cancer

31 March 2020

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A microRNA component of the p53 tumour suppressor network

31 March 2020

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Systemic delivery of tumor suppressor microRNA mimics using a neutral lipid emulsion inhibits lung tumors in mice

31 March 2020

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

31 March 2020

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

31 March 2020

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Genome editing with engineered zinc finger nucleases

31 March 2020

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Breaking the code of DNA binding specificity of TAL-type III effectors

31 March 2020

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A simple cipher governs DNA recognition by TAL effectors

31 March 2020

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A TALE nuclease architecture for efficient genome editing

31 March 2020

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Multiplex genome engineering using CRISPR/Cas systems

31 March 2020

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RNA-guided human genome engineering via Cas9

31 March 2020

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

31 March 2020

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

31 March 2020

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

31 March 2020

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

31 March 2020

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31 March 2020

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Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids

31 March 2020

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Stabilized plasmid-lipid particles containing PEG-diacylglycerols exhibit extended circulation lifetimes and tumor selective gene expression

31 March 2020

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Imidazole groups on a linear, cyclodextrin-containing polycation produce enhanced gene delivery via multiple processes

31 March 2020

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Transferrin-Containing, Cyclodextrin Polymer-Based Particles for Tumor-Targeted Gene Delivery

31 March 2020

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

31 March 2020

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A CRISPR way to engineer the human genome

31 March 2020

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Introduction of liposome-encapsulated SV40 DNA into cells

31 March 2020

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12 December 2020

Polylysine copolymers for gene delivery

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12 December 2020

Clinical applications of microRNAs

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12 December 2020

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

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12 December 2020

Attacking the genome: emerging siRNA nanocarriers from concept to clinic

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12 December 2020

Receptor-mediated gene delivery and expression in vivo.

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12 December 2020

Naked DNA gene transfer in mammalian cells

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12 December 2020

Identifiers

DOI

10.1038/NRG3763

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Dimensions Publication ID

2 August 2017

1022388063

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