Journal ArticleDOI
Nonviral Vectors for Gene Delivery
TLDR
Two nonviral gene delivery systems using either biodegradable poly(D,Llactide-co-glycolide) (PLG) nanoparticles or cell penetrating peptide (CPP) complexes have been designed and studied using A549 human lung epithelial cells.Abstract:
The development of nonviral vectors for safe and efficient gene delivery has been gaining considerable attention recently. An ideal nonviral vector must protect the gene against degradation by nuclease in the extracellular matrix, internalize the plasma membrane, escape from the endosomal compartment, unpackage the gene at some point and have no detrimental effects. In comparison to viruses, nonviral vectors are relatively easy to synthesize, less immunogenic, low in cost, and have no limitation in the size of a gene that can be delivered. Significant progress has been made in the basic science and applications of various nonviral gene delivery vectors; however, the majority of nonviral approaches are still inefficient and often toxic. To this end, two nonviral gene delivery systems using either biodegradable poly(D,Llactide-co-glycolide) (PLG) nanoparticles or cell penetrating peptide (CPP) complexes have been designed and studied using A549 human lung epithelial cells. PLG nanoparticles were optimized for gene delivery by varying particle surface chemistry using different coating materials that adsorb to the particle surface during formation. A variety of cationic coating materials were studied and compared to more conventional surfactants used for PLG nanoparticle fabrication. Nanoparticles (~200 nm) efficiently encapsulated plasmids encoding for luciferase (80-90%) and slowly released the same for two weeks. After a delay, moderate levels of gene expression appeared at day 5 for certain positively charged PLG particles and gene expression was maintained for at least two weeks. In contrast, gene expression mediated by polyethyleneimine (PEI) ended at day 5. PLG particles were also significantly lessread more
Citations
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Journal ArticleDOI
Non-viral vectors for gene-based therapy
Hao Yin,Rosemary Lynn Kanasty,Ahmed A. Eltoukhy,Arturo J. Vegas,J. Robert Dorkin,Daniel G. Anderson +5 more
TL;DR: The biological barriers to gene delivery in vivo are introduced and recent advances in material sciences, nanotechnology and nucleic acid chemistry that have yielded promising non-viral delivery systems are discussed, some of which are currently undergoing testing in clinical trials.
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Functionalizing nanoparticles with biological molecules: developing chemistries that facilitate nanotechnology.
Kim E. Sapsford,W. Russ Algar,Lorenzo Berti,Kelly Boeneman Gemmill,Brendan J. Casey,Eunkeu Oh,Michael H. Stewart,Igor L. Medintz +7 more
TL;DR: Chemistries that Facilitate Nanotechnology Kim E. Sapsford,† W. Russ Algar, Lorenzo Berti, Kelly Boeneman Gemmill,‡ Brendan J. Casey,† Eunkeu Oh, Michael H. Stewart, and Igor L. Medintz .
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Nanochemistry and Nanomedicine for Nanoparticle-based Diagnostics and Therapy
TL;DR: This work presents a new generation of high-performance liquid chromatography platforms for selective separation of Na6(CO3) from Na4(SO4) through Na2SO4 and shows real-world applications in drug discovery and treatment of central nervous system disorders.
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Stimuli-responsive polymeric nanocarriers for the controlled transport of active compounds: Concepts and applications ☆
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Mesoporous Silica Nanoparticles for Intracellular Controlled Drug Delivery
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References
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Journal ArticleDOI
Poly(ethylenimine) and its role in gene delivery
TL;DR: PEI chemistry and the characterization of PEI/DNA complexes used for gene delivery are reviewed and toxicity issues related to PEI transfection are surveyed.
Journal ArticleDOI
Receptor-mediated in vitro gene transformation by a soluble DNA carrier system.
George Y. Wu,C H Wu +1 more
TL;DR: There is evidence that foreign DNA can be specifically delivered to cells by a soluble carrier system that takes advantage of receptor-mediated endocytosis, and that competition by a 10-fold excess of ASOR prevented gene transformation by the ASOR X poly-L-lysine X DNA complex.
Journal ArticleDOI
PEGylated DNA/transferrin–PEI complexes: reduced interaction with blood components, extended circulation in blood and potential for systemic gene delivery
TL;DR: In tumor bearing mice, application of non-PEGylated complexes through the tail vein resulted in reporter gene expression in tail and lung, but severe toxicity was observed in some mice, while PEGylation of the complexes mediated reporter gene transfer to the tumor without significant toxicity.
Journal ArticleDOI
Transvascular delivery of small interfering RNA to the central nervous system
Priti Kumar,Haoquan Wu,Jodi L. McBride,Kyeong Eun Jung,Moon Hee Kim,Beverly L. Davidson,Sang-Kyung Lee,Premlata Shankar,N. Manjunath +8 more
TL;DR: RVG-9R provides a safe and noninvasive approach for the delivery of siRNA and potentially other therapeutic molecules across the blood–brain barrier and afforded robust protection against fatal viral encephalitis in mice.
Journal ArticleDOI
Cell Internalization of the Third Helix of the Antennapedia Homeodomain Is Receptor-independent
Daniele Derossi,Sophie Calvet,Alain Trembleau,Alié Brunissen,Gérard Chassaing,Alain Prochiantz +5 more
TL;DR: The present demonstration, that a reverse helix and a helix composed of D-enantiomers still translocate across biological membranes at 4 and 37°C strongly suggests that the third helix of the homeodomain is internalized by a receptor-independent mechanism.