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
Organelle-specific targeting of polymersomes into the cell nucleus
Christina Zelmer,Ludovit P. Zweifel,Larisa E. Kapinos,Ioana Craciun,Zekiye Pelin Guven,Cornelia G. Palivan,Roderick Y. H. Lim +6 more
TL;DR: The efficacy of polymersomes to deliver encapsulated payloads directly into cell nuclei is demonstrated, as well as the NCT mechanism underlying their selective nuclear uptake, by elucidating their ability to utilize NCT.
Journal ArticleDOI
Biodegradable and Nontoxic Nanogels as Nonviral Gene Delivery Systems
TL;DR: It is found that these cationic glyconanogels can serve as potent gene delivery vectors in hepatocytes and show desirable properties for systemic applications including low toxicity and degradation in acidic environment.
Journal ArticleDOI
Theranostic Prodrug Vesicles for Imaging Guided Codelivery of Camptothecin and siRNA in Synergetic Cancer Therapy
Hongzhong Chen,Huan Jia,Huijun Phoebe Tham,Qiuyu Qu,Pengyao Xing,Jin Zhao,Soo Zeng Fiona Phua,Gang Chen,Yanli Zhao +8 more
TL;DR: A prodrug-based supramolecular amphiphile via the host-guest interaction with better therapeutic performance than free camptothecin is developed, providing an alternative strategy to deliver both prodrug and therapeutic gene.
Journal ArticleDOI
Recent progress in copolymer-mediated siRNA delivery.
TL;DR: Recent progress in copolymer-mediated siRNA delivery is described, including various building blocks for biocompatible copolymers for efficient in vitro si RNA delivery, and a useful basis for addressing the challenges of in vivo siRNA Delivery.
Journal ArticleDOI
Novel self-assembling system based on resorcinarene and cationic surfactant
Ruslan R. Kashapov,Ruslan R. Kashapov,Tatiana N. Pashirova,Sergey V. Kharlamov,Albina Y. Ziganshina,Elena P. Ziltsova,Svetlana S. Lukashenko,Lucia Ya. Zakharova,Lucia Ya. Zakharova,Wolf D. Habicher,Shamil K. Latypov,Alexander I. Konovalov +11 more
TL;DR: Spectrophotometry monitoring of the solubilization of a hydrophobic dye, Orange OT, demonstrated that only the second type of mixed aggregate enriched by DABCO-16 is capable of binding the organic probe, while the mixed system where the surfactant is a minor component shows no binding capacity towards Orange OT.
References
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Journal ArticleDOI
A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine
Otmane Boussif,Frank Lezoualc'h,Maria Antonietta Zanta,Mojgan Mergny,Daniel Scherman,Barbara A. Demeneix,Jean-Paul Behr +6 more
TL;DR: Together, these properties make PEI a promising vector for gene therapy and an outstanding core for the design of more sophisticated devices because its efficiency relies on extensive lysosome buffering that protects DNA from nuclease degradation, and consequent lysOSomal swelling and rupture that provide an escape mechanism for the PEI/DNA particles.
Journal ArticleDOI
Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure
Philip L. Felgner,Thomas R. Gadek,Marilyn Holm,Richard Bolton Roman,Hardy W. Chan,Michael Wenz,Jeffrey P. Northrop,Gordon M. Ringold,Mark Danielsen +8 more
TL;DR: Depending upon the cell line, lipofection is from 5- to greater than 100-fold more effective than either the calcium phosphate or the DEAE-dextran transfection technique.
Journal ArticleDOI
Direct gene transfer into mouse muscle in vivo.
Jon A. Wolff,Robert W. Malone,Phillip Williams,Wang Chong,Gyula Acsadi,Agnes Jani,Philip L. Felgner +6 more
TL;DR: RNA and DNA expression vectors containing genes for chloramphenicol acetyltransferase, luciferase, and beta-galactosidase were separately injected into mouse skeletal muscle in vivo and expression was comparable to that obtained from fibroblasts transfected in vitro under optimal conditions.
Journal ArticleDOI
A new class of polymers: Starburst-dendritic macromolecules
Donald A. Tomalia,H. Baker,James R Dewald,Michael B. Hall,G. Kallos,Steven J. Martin,J. Roeck,J. Ryder,Patrick B. Smith +8 more
TL;DR: Starburst polymers as mentioned in this paper are a class of topological macromolecules which are derived from classical monomers/oligomers by their extraordinary symmetry, high branching and maximized terminal functionality density.