Synthetic delivery systems for intravenous administration of nucleic acids.

At present, there are no intravenously administered nucleic acid-based therapeutics that have been approved for human use. This reflects the difficulties in applying nucleic acid-based drugs: they are nuclease sensitive and have difficulties in reaching their site of action. Important challenges for intravenously administered nucleic acid formulations are the requirements that they can transport the nucleic acids efficiently in the circulation, have the ability to direct nucleic acids to the desired cell type and are able to steer their intracellular processing. Here, we evaluate nanotechnological strategies that improve the pharmacokinetics and colloidal stability of nucleic acids in the bloodstream, focus biodistribution towards the target tissue and facilitate interactions with and trafficking within the desired cell type.

[1]  W. Hennink,et al.  Effect of cationic carriers on the pharmacokinetics and tumor localization of nucleic acids after intravenous administration. , 2007, International journal of pharmaceutics.

[2]  D. Hoekstra,et al.  Cationic lipids, lipoplexes and intracellular delivery of genes. , 2006, Journal of controlled release : official journal of the Controlled Release Society.

[3]  Leaf Huang,et al.  Gene therapy progress and prospects: non-viral gene therapy by systemic delivery , 2006, Gene Therapy.

[4]  Y. Saijoh,et al.  Optimized cationic lipid‐based gene delivery reagents for use in developing vertebrate embryos , 2006, Developmental dynamics : an official publication of the American Association of Anatomists.

[5]  P. Rosenfeld,et al.  Promising new treatments for neovascular age-related macular degeneration , 2006, Expert opinion on investigational drugs.

[6]  Lonnie D Shea,et al.  Design of modular non-viral gene therapy vectors. , 2006, Biomaterials.

[7]  D. Dean,et al.  Intracellular trafficking of plasmids during transfection is mediated by microtubules. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.

[8]  D. Guyer,et al.  Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease , 2006, Nature Reviews Drug Discovery.

[9]  J. Fernandes,et al.  Synthetic and natural polycations for gene therapy: state of the art and new perspectives. , 2006, Current gene therapy.

[10]  Nicholas A Peppas,et al.  Opsonization, biodistribution, and pharmacokinetics of polymeric nanoparticles. , 2006, International journal of pharmaceutics.

[11]  Janos Szebeni,et al.  Complement activation-related pseudoallergy: a new class of drug-induced acute immune toxicity. , 2005, Toxicology.

[12]  M. Kirkham,et al.  Erratum to “Clathrin-independent endocytosis: New insights into caveolae and non-caveolar lipid raft carriers” [Biochim. Biophys. Acta 1744 (2005) 273–286] , 2005 .

[13]  Francis C Szoka,et al.  Designing dendrimers for biological applications , 2005, Nature Biotechnology.

[14]  J. Kos,et al.  Nanoscale polymer carriers to deliver chemotherapeutic agents to tumours , 2005, Expert opinion on biological therapy.

[15]  S Moein Moghimi,et al.  Low and high molecular weight poly(l‐lysine)s/poly(l‐lysine)–DNA complexes initiate mitochondrial‐mediated apoptosis differently , 2005, FEBS letters.

[16]  M. Türk,et al.  Intelligent polymers as nonviral vectors , 2005, Gene Therapy.

[17]  M. Monsigny,et al.  Potocytosis and Cellular Exit of Complexes as Cellular Pathways for Gene Delivery by Polycations , 2005 .

[18]  R. Schiffelers,et al.  Liposomal targeting of angiogenic vasculature. , 2005, Current drug delivery.

[19]  S. Hart.  Lipid carriers for gene therapy. , 2005, Current drug delivery.

[20]  T. Allen,et al.  Ligand-targeted liposomes for cancer treatment. , 2005, Current drug delivery.

[21]  M. Bergström,et al.  Biodistribution of 68Ga-labelled phosphodiester, phosphorothioate, and 2'-O-methyl phosphodiester oligonucleotides in normal rats. , 2005, European journal of pharmaceutical sciences : official journal of the European Federation for Pharmaceutical Sciences.

[22]  M. Conese,et al.  Role of clathrin- and caveolae-mediated endocytosis in gene transfer mediated by lipo- and polyplexes. , 2005, Molecular therapy : the journal of the American Society of Gene Therapy.

[23]  D. Hoekstra,et al.  Sunfish cationic amphiphiles: toward an adaptative lipoplex morphology. , 2005, Journal of the American Chemical Society.

[24]  H. Nauwynck,et al.  Binding and entry characteristics of porcine circovirus 2 in cells of the porcine monocytic line 3D4/31. , 2005, The Journal of general virology.

[25]  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.

[26]  Roy Bicknell,et al.  Tumour vascular targeting , 2005, Nature Reviews Cancer.

[27]  N. Hud,et al.  Toroidal DNA condensates: unraveling the fine structure and the role of nucleation in determining size. , 2005, Annual review of biophysics and biomolecular structure.

[28]  R. Langer,et al.  Exploring polyethylenimine‐mediated DNA transfection and the proton sponge hypothesis , 2005, The journal of gene medicine.

[29]  Qing Ge,et al.  Full deacylation of polyethylenimine dramatically boosts its gene delivery efficiency and specificity to mouse lung. , 2005, Proceedings of the National Academy of Sciences of the United States of America.

[30]  D. Burgess,et al.  DNA-based therapeutics and DNA delivery systems: A comprehensive review , 2005, The AAPS Journal.

[31]  Feng Liu,et al.  Implications of pharmacokinetic behavior of lipoplex for its inflammatory toxicity. , 2005, Advanced drug delivery reviews.

[32]  R. Mahato.  Water insoluble and soluble lipids for gene delivery. , 2005, Advanced drug delivery reviews.

[33]  G. Lukács,et al.  Intracellular routing of plasmid DNA during non-viral gene transfer. , 2005, Advanced drug delivery reviews.

[34]  M. Mano,et al.  Cationic liposomes for gene delivery , 2005, Expert opinion on drug delivery.

[35]  Mayank M. Patel,et al.  Contribution of hydrophobicity to thermodynamics of ligand-DNA binding and DNA collapse. , 2005, Biophysical journal.

[36]  M. Ogris,et al.  Specific targets in tumor tissue for the delivery of therapeutic genes. , 2005, Current medicinal chemistry. Anti-cancer agents.

[37]  L. Brannon-Peppas,et al.  Nanoparticle and targeted systems for cancer therapy. , 2004, Advanced drug delivery reviews.

[38]  D. Fischer,et al.  Effect of poly(ethylene imine) molecular weight and pegylation on organ distribution and pharmacokinetics of polyplexes with oligodeoxynucleotides in mice. , 2004, Drug metabolism and disposition: the biological fate of chemicals.

[39]  A. Villaverde,et al.  Modular protein engineering for non-viral gene therapy. , 2004, Trends in biotechnology.

[40]  M. Yessine,et al.  Membrane-destabilizing polyanions: interaction with lipid bilayers and endosomal escape of biomacromolecules. , 2004, Advanced drug delivery reviews.

[41]  N. Hud,et al.  Evidence that both kinetic and thermodynamic factors govern DNA toroid dimensions: effects of magnesium(II) on DNA condensation by hexammine cobalt(III). , 2004, Biochemistry.

[42]  P. Cullis,et al.  Drug Delivery Systems: Entering the Mainstream , 2004, Science.

[43]  A. Kichler.  Gene transfer with modified polyethylenimines , 2004, The journal of gene medicine.

[44]  Daniel Scherman,et al.  Physicochemical optimisation of plasmid delivery by cationic lipids , 2004, The journal of gene medicine.

[45]  D. Crommelin,et al.  Steric stabilization of poly(2‐(dimethylamino)ethyl methacrylate)‐based polyplexes mediates prolonged circulation and tumor targeting in mice , 2004, The journal of gene medicine.

[46]  J Szebeni,et al.  Stealth liposomes and long circulating nanoparticles: critical issues in pharmacokinetics, opsonization and protein-binding properties. , 2003, Progress in lipid research.

[47]  L. Pelkmans,et al.  Insider information: what viruses tell us about endocytosis. , 2003, Current opinion in cell biology.

[48]  T. Yoshida,et al.  ε-Poly-l-lysine: microbial production, biodegradation and application potential , 2003, Applied Microbiology and Biotechnology.

[49]  D. Fischer,et al.  Low-molecular-weight polyethylenimine as a non-viral vector for DNA delivery: comparison of physicochemical properties, transfection efficiency and in vivo distribution with high-molecular-weight polyethylenimine. , 2003, Journal of controlled release : official journal of the Controlled Release Society.

[50]  Jens Kurreck,et al.  Antisense technologies. Improvement through novel chemical modifications. , 2003, European journal of biochemistry.

[51]  Jayanth Panyam,et al.  Biodegradable nanoparticles for drug and gene delivery to cells and tissue. , 2003, Advanced drug delivery reviews.

[52]  Marie Carrière,et al.  NLS bioconjugates for targeting therapeutic genes to the nucleus. , 2003, Advanced drug delivery reviews.

[53]  S. Akira,et al.  Recognition of pathogen-associated molecular patterns by TLR family. , 2003, Immunology letters.

[54]  M. Ariatti,et al.  A Note on Poly-L-Lysine-Mediated Gene Transfer in HeLa Cells , 2003, Drug delivery.

[55]  Mary J Cloninger,et al.  Biological applications of dendrimers. , 2002, Current opinion in chemical biology.

[56]  Y. Oh,et al.  Polyethylenimine-mediated cellular uptake, nucleus trafficking and expression of cytokine plasmid DNA , 2002, Gene Therapy.

[57]  Chantal Pichon,et al.  Histidine Containing Peptides and Polypeptides as Nucleic Acid Vectors , 2002, Somatic cell and molecular genetics.

[58]  Jindrich Kopecek,et al.  Prospects for cationic polymers in gene and oligonucleotide therapy against cancer. , 2002, Advanced drug delivery reviews.

[59]  K. Ulbrich,et al.  Modification of pLL/DNA complexes with a multivalent hydrophilic polymer permits folate‐mediated targeting in vitro and prolonged plasma circulation in vivo , 2002, The journal of gene medicine.

[60]  Y. Barenholz,et al.  Lipoplex-induced hemagglutination: potential involvement in intravenous gene delivery , 2002, Gene Therapy.

[61]  G. Whittaker,et al.  Dissecting virus entry via endocytosis. , 2002, The Journal of general virology.

[62]  D. Fischer,et al.  The Structure of PEG-Modified Poly(Ethylene Imines) Influences Biodistribution and Pharmacokinetics of Their Complexes with NF-κB Decoy in Mice , 2002, Pharmaceutical Research.

[63]  M. Carbone,et al.  The Role of Environmental Carcinogens, Viruses, and Genetic , 2002, Cancer biology & therapy.

[64]  J. Dennig,et al.  Gene transfer into eukaryotic cells using activated polyamidoamine dendrimers. , 2002, Journal of biotechnology.

[65]  G. Lukács,et al.  Intracellular barriers to non-viral gene transfer. , 2002, Current gene therapy.

[66]  Kenneth A Howard,et al.  Importance of lateral and steric stabilization of polyelectrolyte gene delivery vectors for extended systemic circulation. , 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

[67]  P. Griffiths,et al.  Fomivirsen for the treatment of cytomegalovirus retinitis. , 2002, American journal of ophthalmology.

[68]  Qingrong Chen,et al.  Optimal transfection with the HK polymer depends on its degree of branching and the pH of endocytic vesicles. , 2002, Nucleic acids research.

[69]  N. Dias,et al.  Antisense oligonucleotides: basic concepts and mechanisms. , 2002, Molecular cancer therapeutics.

[70]  D. Hoekstra,et al.  Molecular Shape of the Cationic Lipid Controls the Structure of Cationic Lipid/Dioleylphosphatidylethanolamine-DNA Complexes and the Efficiency of Gene Delivery* , 2001, The Journal of Biological Chemistry.

[71]  S. Carotta,et al.  Different behavior of branched and linear polyethylenimine for gene delivery in vitro and in vivo , 2001, The journal of gene medicine.

[72]  M. Hashida,et al.  Hepatic uptake and gene expression mechanisms following intravenous administration of plasmid DNA by conventional and hydrodynamics-based procedures. , 2001, The Journal of pharmacology and experimental therapeutics.

[73]  E. Wagner,et al.  Tumor targeting with surface-shielded ligand--polycation DNA complexes. , 2001, Journal of controlled release : official journal of the Controlled Release Society.

[74]  A. Schatzlein,et al.  Non-viral vectors in cancer gene therapy: principles and progress , 2001, Anti-cancer drugs.

[75]  S. Stass,et al.  Branched co-polymers of histidine and lysine are efficient carriers of plasmids. , 2001, Nucleic acids research.

[76]  C. Férec,et al.  Cation substitution in cationic phosphonolipids: a new concept to improve transfection activity and decrease cellular toxicity. , 2000, Journal of medicinal chemistry.

[77]  E. W. Meijer,et al.  Dendrimers: relationship between structure and biocompatibility in vitro, and preliminary studies on the biodistribution of 125I-labelled polyamidoamine dendrimers in vivo. , 2000, Journal of controlled release : official journal of the Controlled Release Society.

[78]  M. Gottikh,et al.  Physico-chemical and biological properties of antisense phosphodiester oligonucleotides with various secondary structures. , 1999, Nucleosides & nucleotides.

[79]  Wagner,et al.  Application of membrane-active peptides for nonviral gene delivery. , 1999, Advanced drug delivery reviews.

[80]  M. Ogris,et al.  PEGylated DNA/transferrin–PEI complexes: reduced interaction with blood components, extended circulation in blood and potential for systemic gene delivery , 1999, Gene Therapy.

[81]  H. O'brodovich,et al.  Metabolic instability of plasmid DNA in the cytosol: a potential barrier to gene transfer , 1999, Gene Therapy.

[82]  D. Hanahan,et al.  Cationic liposomes target angiogenic endothelial cells in tumors and chronic inflammation in mice. , 1998, The Journal of clinical investigation.

[83]  P. Klotman,et al.  Identification and characterization of a cell membrane nucleic acid channel. , 1998, Proceedings of the National Academy of Sciences of the United States of America.

[84]  R K Jain,et al.  Vascular permeability in a human tumor xenograft: molecular size dependence and cutoff size. , 1995, Cancer research.

[85]  H. Farhood,et al.  The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer. , 1995, Biochimica et biophysica acta.

[86]  Kari Syrjänen,et al.  Comparison of Cell Proliferation and Toxicity Assays Using Two Cationic Liposomes , 1994, Pharmaceutical Research.

[87]  Robert G Parton,et al.  Clathrin-independent endocytosis: new insights into caveolae and non-caveolar lipid raft carriers. , 2005, Biochimica et biophysica acta.

[88]  S. May,et al.  Modeling of cationic lipid-DNA complexes. , 2004, Current medicinal chemistry.

[89]  C. van Nostrum,et al.  Polymer Side-Chain Degradation as a Tool to Control the Destabilization of Polyplexes , 2004, Pharmaceutical Research.

[90]  J. Kurreck.  Improvement through novel chemical modifications , 2003 .

[91]  I. Maclachlan,et al.  Stabilized plasmid-lipid particles: a systemic gene therapy vector. , 2002, Methods in enzymology.

[92]  A. Krieg,et al.  CpG motifs in bacterial DNA and their immune effects. , 2002, Annual review of immunology.

[93]  L. Seymour,et al.  Triggered intracellular activation of disulfide crosslinked polyelectrolyte gene delivery complexes with extended systemic circulation in vivo , 2001, Gene Therapy.

[94]  M. Garnett,et al.  Gene-delivery systems using cationic polymers. , 1999, Critical reviews in therapeutic drug carrier systems.

[95]  H. Ohshima,et al.  Determination of the thickness of the fixed aqueous layer around polyethyleneglycol-coated liposomes. , 1995, Journal of drug targeting.