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
More filters
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.
Journal ArticleDOI
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 .
Journal ArticleDOI
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.
Journal ArticleDOI
Stimuli-responsive polymeric nanocarriers for the controlled transport of active compounds: Concepts and applications ☆
TL;DR: This review gives a brief overview about some types of stimuli-responsive nanocarriers with the main focus on organic polymer-based systems.
Journal ArticleDOI
Mesoporous Silica Nanoparticles for Intracellular Controlled Drug Delivery
TL;DR: The latest research on the pathways of entry into live mammalian and plant cells together with intracellular trafficking are described, and the current research progress on the biocompatibility of this material in vitro and in vivo is discussed.
References
More filters
Journal ArticleDOI
Endosomal Escape of Polymeric Gene Delivery Complexes Is Not Always Enhanced by Polymers Buffering at Low pH
Arjen M. Funhoff,Cornelus F. van Nostrum,Gerben A. Koning,N.M.E. Schuurmans-Nieuwenbroek,Daan J.A. Crommelin,Wim E. Hennink +5 more
TL;DR: It was shown that pDAMA is able to condense DNA in small particles with a surface charge depending on the polymer/DNA ratio and has a substantial lower toxicity than other polymeric transfectants.
Journal ArticleDOI
Ultrasonic degradation of schizophyllan, an antitumor polysaccharide produced by Schizophyllum commune fries
TL;DR: End-group analysis by using radioisotope-labeled glucans suggests that ultrasonic degradation occurs mainly by cleavage of glycosidic bonds of the main chain of schizophyllan, and there is no anomalous linkage sensitive to periodate oxidation.
Journal ArticleDOI
A novel biodegradable gene carrier based on polyphosphoester.
Journal ArticleDOI
Different mechanisms for cellular internalization of the HIV-1 Tat-derived cell penetrating peptide and recombinant proteins fused to Tat.
TL;DR: It is demonstrated here that the short Tat CPP is taken up by a route that does not involve the HS proteoglycans, which is a major limiting step for the cellular delivery of macromolecules.
Journal ArticleDOI
ExGen 500 is an efficient vector for gene delivery to lung epithelial cells in vitro and in vivo.
Stefano Ferrari,Enrico Moro,Andrea Pettenazzo,Jean-Paul Behr,Franco Zacchello,Maurizio Scarpa +5 more
TL;DR: In vivo ExGen 500 was able to mediate gene transfer into both newborn and adult rabbit lungs with comparable efficiencies, and within 1 week after instillation, transgene expression decreased by two orders of magnitude.