Gene delivery
Vectors and Gene Delivery to the Retina
... Retinal gene therapy aims to target these neurons in an efficient, safe, and specific way so that ther- apeutic gene expression can bring on ...between gene interaction networks that regulate complex ...
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Degradable Terpolymers with Alkyl Side Chains Demonstrate Enhanced Gene Delivery Potency and Nanoparticle Stability
... polyplexes formed from D60-122 polymer lacking alkyl side chains grew 50% in diameter, in contrast to <10% growth for those formed from D60-122 terpolymers synthesized with C12 feed ratios at or above 20 mol%. To ...
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Gene delivery to pancreatic exocrine cells in vivo and in vitro
... efficient gene delivery into pancreatic acinar cells, we have investigated different viral and non-viral vectors in vitro and in ...of gene transfer with lipo- fectamine 2000 ...
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Chemical modification of the adeno-associated virus capsid to improve gene delivery
... Gene delivery vectors based on adeno-associated virus (AAV) are highly promising due to several desirable features of this parent virus, including a lack of pathogenicity, e fficient infection of dividing and ...
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Systemic gene delivery to the central nervous system using Adeno-associated virus
... However, gene therapy for neurodegenerative disorders, which are mainly adult-onset diseases, would ideally occur in adult, unless familial diseases or cases of otherwise sporadic diseases are ...systemic ...
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Ultrasound and microbubble-assisted gene delivery in Achilles tendons: long lasting gene expression and restoration of fibromodulin KO phenotype
... fibromodulin gene in fibromodulin KO ...this gene delivery method is promising for clinical applications aimed at modulating healing or restoring a degenerative tendon while offering great promise for ...
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Tailoring the surface of a gene delivery vector with carboxymethylated dextran: a systematic analysis
... model gene delivery nanoparticle, we chose branched polyethylenimine (bPEI)/plasmid DNA (pDNA) complexes, referred here as polyplexes, that are known to feature high transfection capabilities in vitro, but ...
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Electromediated formation of DNA complexes with cell membranes and its consequences for gene delivery
... the order of seconds. Here, cells are electropermeabilized in the presence of plasmid DNA and monitored with a temporal resolution of 2 ms. This allows us to show that during the first pulse application, plasmid ...
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Ultrasound and microbubble-assisted gene delivery: recent advances and ongoing challenges
... ultrasound parameters for cardiac gene delivery of adenoviral or plasmid 962. deoxyribonucleic acid by ultrasound-targeted microbubble destruction[r] ...
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Revisiting Cationic Phosphorus Dendrimers as a Nonviral Vector for Optimized Gene Delivery Toward Cancer Therapy Applications
... 20. on l es C ou iu h al , H.; Tomás, H.; Shi, X. Y., RGD peptide-modified dendrimer-entrapped gold nanoparticles enable highly efficient and specific gene delivery to stem cells. ACS Appl. Mater. ...
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Aggregation of Synthetic Gene Delivery Vectors Enhance the Cellular Association and Uptake for in vitro Transfection
... In this study, the correlation of physicochemical properties of polymer- based synthetic gene delivery vectors (polyplexes) with cellular association as the first barrier for in vitr[r] ...
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Efficient gene delivery by sonoporation Is associated with microbubble entry into cells and the clathrin-dependent endocytosis pathway
... Efficient gene delivery by sonoporation Is associated with microbubble entry into cells and the clathrin-dependent endocytosis pathway.8. Title: Efficient gene delivery by sonoporati[r] ...
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Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain
... combining gene therapy with an endoscopic approach to enable tracking of exo-AAV spread, and (2) exo-AAVs allow for widespread, long-term gene expression in the CNS, supporting the use of exo-AAVs as an ef ...
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Coupling of RAFT polymerization and chemoselective post-modifications of elastin-like polypeptides for the synthesis of gene delivery hybrid vectors
... acids delivery due to the possibility of modulating and quantifying the number of cationic ...and delivery were synthetized through the coupling of RAFT polymerization and biorthogonal chemistry of ELPs ...
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Peptides derived from the C-terminal domain of HIV-1 Viral Protein R in lipid bilayers: Structure, membrane positioning and gene delivery
... 3. Results 3.1. Plasmid DNA transfection Previously, we reported that the C-terminal domain of Vpr and in particular sub-fragments of Vpr52-96, are very robust transfection agents [17, 18]. According to NMR studies ...
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Chloroquine-enhanced gene delivery mediated by carbon nanotubes
... 100 lM) [36–38] ; and so was highly unlikely to be due to leeching out of the chloroquine prior to entry of CNTs inside cells. The other forms of DWCNTs assayed for optimizing chloroquine loading were also tested to ...
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PEGylated quaternized copolymer/DNA complexes for gene delivery
... The main aim of this present study was to improve the colloidal stability and in vivo biodistribution of the novel gene delivery systems based on ε-caprolactone and quaternized ε-caprolactone (Detrembleur ...
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Stem cells as gene delivery system to create cardiac biological pacemaker
... Adult human mesenchymal stem cells (hMSCs) are attractive for gene delivery applications [5]. The most important point is that these cells are immunoprivileged, in other words, they might not elicit the ...
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Noninvasive gene delivery to foveal cones for vision restoration
... to gene therapy, we first searched for promoters that are able to afford high-level transgene expres- sion in cones without leading to off-target expression in neighboring ...provide gene delivery to ...
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Ultrasound and microbubble-assisted gene delivery: insights for intracellular mechanism
... microbubble-assisted gene delivery: insights for intracellular mechanism Ultrasound exposure system for high-speed imaging For fast camera imaging experiments, the transducer was placed in a custom-made ...
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