Recent trends in non‐viral vector‐mediated gene delivery

Nucleic acids‐based next generation biopharmaceuticals (i.e., pDNA, oligonucleotides, short interfering RNA) are potential pioneering materials to cope with various incurable diseases. However, several biological barriers present a challenge for efficient gene delivery. On the other hand, developments in nanotechnology now offer numerous non‐viral vectors that have been fabricated and found capable of transmitting the biopharmaceuticals into the cell and even into specific subcellular compartments like mitochondria. This overview illustrates cellular barriers and current status of non‐viral gene vectors, i.e., lipoplexes, liposomes, polyplexes, and nanoparticles, to relocate therapeutic DNA‐based nanomedicine into the target cell. Despite the awesome impact of physical methods (i.e., ultrasound, electroporation), chemical methods have been shown to accomplish high‐level and safe transgene expression. Further comprehension of barriers and the mechanism of cellular uptake will facilitate development of nucleic acids‐based nanotherapy for alleviation of various disorders.

[1]  Leaf Huang,et al.  Nonviral methods for siRNA delivery. , 2009, Molecular pharmaceutics.

[2]  Chantal Pichon,et al.  Chemical vectors for gene delivery: a current review on polymers, peptides and lipids containing histidine or imidazole as nucleic acids carriers , 2009, British journal of pharmacology.

[3]  Enrico Gratton,et al.  Efficient escape from endosomes determines the superior efficiency of multicomponent lipoplexes. , 2009, The journal of physical chemistry. B.

[4]  Daniel G. Anderson,et al.  Polymeric Materials for Gene Delivery and DNA Vaccination , 2009, Advanced materials.

[5]  Meredith A Mintzer,et al.  Nonviral vectors for gene delivery. , 2009, Chemical reviews.

[6]  J. Engbersen,et al.  Effect of chemical functionalities in poly(amido amine)s for non-viral gene transfection. , 2008, Journal of controlled release : official journal of the Controlled Release Society.

[7]  T. H. Dung,et al.  Preparation and biophysical characterization of pluronic F127-dendrimer conjugate as a delivery agent of antisense oligonucleotides. , 2008, Journal of nanoscience and nanotechnology.

[8]  J. Ruysschaert,et al.  Cationic liposomal lipids: from gene carriers to cell signaling. , 2008, Progress in lipid research.

[9]  Y. Maitani,et al.  Effect of sugars on storage stability of lyophilized liposome/DNA complexes with high transfection efficiency. , 2008, International journal of pharmaceutics.

[10]  J. Y. Lee,et al.  Polyethyleneimine-mediated gene delivery into human adipose derived stem cells. , 2008, Biomaterials.

[11]  M. de la Fuente,et al.  Novel hyaluronic acid-chitosan nanoparticles for ocular gene therapy. , 2008, Investigative ophthalmology & visual science.

[12]  Guping Tang,et al.  Low Generation Polypropylenimine Dendrimer Graft β-cyclodextrin: An Efficient Vector for Gene Delivery System , 2008, Technology in cancer research & treatment.

[13]  Wim E Hennink,et al.  Biodegradable polymers as non-viral carriers for plasmid DNA delivery. , 2008, Journal of controlled release : official journal of the Controlled Release Society.

[14]  S. Kasturi,et al.  Enhancing polysaccharide-mediated delivery of nucleic acids through functionalization with secondary and tertiary amines. , 2008, Current topics in medicinal chemistry.

[15]  Suzie H Pun,et al.  Extracellular barriers to in Vivo PEI and PEGylated PEI polyplex-mediated gene delivery to the liver. , 2008, Bioconjugate chemistry.

[16]  C. Cho,et al.  Urocanic acid-modified chitosan-mediated PTEN delivery via aerosol suppressed lung tumorigenesis in K-rasLA1 mice , 2008, Cancer Gene Therapy.

[17]  B. Houk,et al.  Kinetic modeling of plasmid DNA degradation in rat plasma , 1999, AAPS PharmSci.

[18]  R. Zhuo,et al.  Dendrimer/DNA complexes encapsulated in a water soluble polymer and supported on fast degrading star poly(DL-lactide) for localized gene delivery. , 2007, Journal of controlled release : official journal of the Controlled Release Society.

[19]  M. Schoenfisch,et al.  Cytotoxicity of polypropylenimine dendrimer conjugates on cultured endothelial cells. , 2007, Biomacromolecules.

[20]  H. Uludaǧ,et al.  A comparative evaluation of poly-L-lysine-palmitic acid and Lipofectamine 2000 for plasmid delivery to bone marrow stromal cells. , 2007, Biomaterials.

[21]  Jo Wixon,et al.  Gene therapy clinical trials worldwide to 2007—an update , 2007, The journal of gene medicine.

[22]  M. Ogris,et al.  A dimethylmaleic acid–melittin‐polylysine conjugate with reduced toxicity, pH‐triggered endosomolytic activity and enhanced gene transfer potential , 2007, The journal of gene medicine.

[23]  Xu-li Wang,et al.  Synthesis, characterization, and gene delivery of poly-L-lysine octa(3-aminopropyl)silsesquioxane dendrimers: nanoglobular drug carriers with precisely defined molecular architectures. , 2007, Molecular pharmaceutics.

[24]  K. Gupta,et al.  Engineered Polyallylamine Nanoparticles for Efficient In Vitro Transfection , 2007, Pharmaceutical Research.

[25]  N. Enomoto,et al.  Genetic manipulation of sinusoidal endothelial cells , 2001, Journal of gastroenterology and hepatology.

[26]  P. Artursson,et al.  Targeted gene delivery with trisaccharide-substituted chitosan oligomers in vitro and after lung administration in vivo. , 2006, Journal of controlled release : official journal of the Controlled Release Society.

[27]  R. Chandra,et al.  Novel polyallylamine-dextran sulfate-DNA nanoplexes: highly efficient non-viral vector for gene delivery. , 2006, International journal of pharmaceutics.

[28]  V. Shenoy,et al.  Role of calcium in gene delivery , 2006, Expert opinion on drug delivery.

[29]  Toshinori Sato,et al.  Lactosylated chitosan for DNA delivery into hepatocytes: the effect of lactosylation on the physicochemical properties and intracellular trafficking of pDNA/chitosan complexes. , 2006, Bioconjugate chemistry.

[30]  L. Young,et al.  Viral gene therapy strategies: from basic science to clinical application , 2006, The Journal of pathology.

[31]  S Moein Moghimi,et al.  A two-stage poly(ethylenimine)-mediated cytotoxicity: implications for gene transfer/therapy. , 2005, Molecular therapy : the journal of the American Society of Gene Therapy.

[32]  Dexi Liu,et al.  1110. Selective Chemical Modification on Polyethylenimine and Its Effects on Transfection Efficiency and Cytotoxicity , 2005 .

[33]  Sung Wan Kim,et al.  Polyethylenimine with acid-labile linkages as a biodegradable gene carrier. , 2005, Journal of controlled release : official journal of the Controlled Release Society.

[34]  Vladimir P Torchilin,et al.  Intracellular delivery of large molecules and small particles by cell-penetrating proteins and peptides. , 2005, Advanced drug delivery reviews.

[35]  Clive J Roberts,et al.  Preferential liver gene expression with polypropylenimine dendrimers. , 2005, Journal of controlled release : official journal of the Controlled Release Society.

[36]  A. Jones,et al.  Intracellular trafficking pathways and drug delivery: fluorescence imaging of living and fixed cells. , 2005, Advanced drug delivery reviews.

[37]  Ann Logan,et al.  Barriers to Gene Delivery Using Synthetic Vectors. , 2005, Advances in genetics.

[38]  Mansoor M. Amiji,et al.  Polymeric Gene Delivery : Principles and Applications , 2004 .

[39]  F André,et al.  DNA electrotransfer: its principles and an updated review of its therapeutic applications , 2004, Gene therapy.

[40]  Mark E. Davis,et al.  Cyclodextrin-modified polyethylenimine polymers for gene delivery. , 2004, Bioconjugate chemistry.

[41]  A. Domb,et al.  Current developments in gene transfection agents. , 2004, Current drug delivery.

[42]  C. Magin-Lachmann,et al.  In vitro and in vivo delivery of intact BAC DNA – comparison of different methods , 2004, The journal of gene medicine.

[43]  R. Mahato,et al.  Cationic Lipid-Based Gene Delivery Systems: Pharmaceutical Perspectives , 1997, Pharmaceutical Research.

[44]  Fred D. Ledley,et al.  Pharmaceutical Approach to Somatic Gene Therapy , 1996, Pharmaceutical Research.

[45]  Khursheed Anwer,et al.  Polyvinyl Derivatives as Novel Interactive Polymers for Controlled Gene Delivery to Muscle , 1996, Pharmaceutical Research.

[46]  M. Hashida,et al.  The Fate of Plasmid DNA After Intravenous Injection in Mice: Involvement of Scavenger Receptors in Its Hepatic Uptake , 1995, Pharmaceutical Research.

[47]  I. Zuhorn,et al.  Size-dependent internalization of particles via the pathways of clathrin- and caveolae-mediated endocytosis. , 2004, The Biochemical journal.

[48]  I. Srivastava,et al.  Gene Vaccines , 2003, Annals of Internal Medicine.

[49]  Jung-Ki Park,et al.  Effect of polyethylene glycol on gene delivery of polyethylenimine. , 2003, Biological & pharmaceutical bulletin.

[50]  D. Wirtz,et al.  Efficient active transport of gene nanocarriers to the cell nucleus , 2003, Proceedings of the National Academy of Sciences of the United States of America.

[51]  B. Gansbacher Report of a second serious adverse event in a clinical trial of gene therapy for X‐linked severe combined immune deficiency (X‐SCID) , 2003, The journal of gene medicine.

[52]  Kevin E Healy,et al.  Nanoparticulate DNA packaging using terpolymers of poly(lysine-g-(lactide-b-ethylene glycol)). , 2003, Bioconjugate chemistry.

[53]  J. Hagstrom,et al.  Recharging cationic DNA complexes with highly charged polyanions for in vitro and in vivo gene delivery , 2003, Gene Therapy.

[54]  A. Klibanov,et al.  Enhancing polyethylenimine's delivery of plasmid DNA into mammalian cells , 2002, Proceedings of the National Academy of Sciences of the United States of America.

[55]  V. Labhasetwar,et al.  Size-dependency of nanoparticle-mediated gene transfection: studies with fractionated nanoparticles. , 2002, International journal of pharmaceutics.

[56]  J Henke,et al.  Magnetofection: enhancing and targeting gene delivery by magnetic force in vitro and in vivo , 2002, Gene Therapy.

[57]  M. Hashida,et al.  Cell-specific delivery of genes with glycosylated carriers. , 2001, Advanced drug delivery reviews.

[58]  J. Wolff,et al.  Efficient expression of naked dna delivered intraarterially to limb muscles of nonhuman primates. , 2001, Human gene therapy.

[59]  J. Hubbell,et al.  Blocking adhesion to cell and tissue surfaces by the chemisorption of a poly-L-lysine-graft-(poly(ethylene glycol); phenylboronic acid) copolymer. , 2000, Biomacromolecules.

[60]  Chang-qing Yang,et al.  Glyco-poly-l-lysine is better than liposomal delivery of exogenous genes to rat of liver. , 2000, World journal of gastroenterology.

[61]  D. Lauffenburger,et al.  Vector unpacking as a potential barrier for receptor-mediated polyplex gene delivery. , 2000, Biotechnology and bioengineering.

[62]  L. Véron,et al.  Synthesis of polyallylamine derivatives and their use as gene transfer vectors in vitro. , 1999, Bioconjugate chemistry.

[63]  A. Mikos,et al.  Poly(ethylenimine) and its role in gene delivery. , 1999, Journal of controlled release : official journal of the Controlled Release Society.

[64]  D. Hwang,et al.  Lactoferrin as a gene delivery vehicle to hepatocytes , 1997, Experimental & Molecular Medicine.

[65]  D. Scherman,et al.  A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. , 1995, Proceedings of the National Academy of Sciences of the United States of America.

[66]  B. Lebleu,et al.  Intracellular distribution of microinjected antisense oligonucleotides. , 1991, Proceedings of the National Academy of Sciences of the United States of America.

[67]  J. Northrop,et al.  Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. , 1987, Proceedings of the National Academy of Sciences of the United States of America.

[68]  James R. Dewald,et al.  A New Class of Polymers: Starburst-Dendritic Macromolecules , 1985 .

[69]  R. Hoffman,et al.  Binding and entrapment of high molecular weight DNA by lecithin liposomes , 1978, FEBS letters.