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
NanoRNP Overcomes Tumor Heterogeneity in Cancer Treatment
Qi Liu,Jinquan Cai,Yadan Zheng,Yanli Tan,Yunfei Wang,Zhanzhan Zhang,Chunxiong Zheng,Yu Zhao,Chaoyong Liu,Yingli An,Chuanlu Jiang,Linqi Shi,Chunsheng Kang,Yang Liu +13 more
TL;DR: Considering the close relationship between tumor heterogeneity and cancer progression, resistance to therapy, and recurrences, nanoRNP provides a feasible strategy to overcome tumor heterogeneity in the development of more advanced cancer therapy against malignant tumors.
Journal ArticleDOI
Porous silicon microparticles for delivery of siRNA therapeutics.
Jianliang Shen,Xiaoyan Wu,Yeonju Lee,Joy Wolfram,Zhizhou Yang,Zong-Wan Mao,Mauro Ferrari,Haifa Shen,Haifa Shen +8 more
TL;DR: A protocol for the preparation and use of a biocompatible and effective siRNA delivery system consisting of polyethylenimine and arginine-grafted porous silicon microparticles that provides a means for protecting and internalizing siRNA, without causing cytotoxicity.
Journal ArticleDOI
Functionalization of SF/HAP Scaffold with GO-PEI-miRNA inhibitor Complexes to Enhance Bone Regeneration through Activating Transcription Factor 4
Lingling Ou,Yong Lan,Zhiqiang Feng,Feng Longbao,Junjie Yang,Yu Liu,Liming Bian,Jiali Tan,Renfa Lai,Rui Guo +9 more
TL;DR: This technology is used to increase osteogenic differentiation and mineralized bone formation in bone defects, which helps to achieve cell-free scaffold-based miRNA-inhibitor therapy for bone tissue engineering.
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Recent review of the effect of nanomaterials on stem cells
TL;DR: It is expected that the enhanced understanding of NM-stem cell interactions will facilitate biomaterial design for stem-cell-therapy and regenerative medicine applications.
Book ChapterDOI
Nonviral vectors: we have come a long way.
Tyler J. Goodwin,Leaf Huang +1 more
TL;DR: This chapter will provide a brief overview on recent advances in the field of nonviral vectors for gene therapy as well as point out some novel vectors that have assisted in the extraordinary growth ofnonviral gene Therapy as the authors know it today.
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.