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Original Article

Non-invasive gene targeting to the fetal brain after intravenous administration and transplacental transfer of plasmid DNA using PEGylated immunoliposomes

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Pages 58-67 | Received 09 Mar 2015, Accepted 24 May 2015, Published online: 02 Jul 2015

References

  • Mok KW, Lam AM Cullis PR. Stabilized plasmid-lipid particles: factors influencing plasmid entrapment and transfection properties. Biochim Biophys Acta 1999;1419:137–50
  • Shi N, Pardridge WM. Noninvasive gene targeting to the brain. Proc Natl Acad Sci USA 2000;97:7567–72
  • Matsui H, Johnson LG, Randell SH, Boucher RC. Loss of binding and entry of liposome-DNA complexes decreases transfection efficiency in differentiated airway epithelial cells. J Biol Chem 1997;272:1117–26
  • Barron LG, Uyechi LS, SzokaJr, FC. Cationic lipids are essential for gene delivery mediated by intravenous administration of lipoplexes. Gene Ther 1999;6:1179–83
  • Osaka G, Carey K, Cuthbertson A, et al. Pharmacokinetics, tissue distribution, and expression efficiency of plasmid [33P]DNA following intravenous administration of DNA/cationic lipid complexes in mice: use of a novel radionuclide approach. J Pharm Sci 1996;85:612–18
  • Culver KW, Ram Z, Wallbridge S, et al. In vivo gene transfer with retroviral vector-producer cells for treatment of experimental brain tumors. Science 1992;256:1550–2
  • Nilaver G, Muldoon LL, Kroll RA, et al. Delivery of herpesvirus and adenovirus to nude rat intracerebral tumors after osmotic blood–brain barrier disruption. Proc Natl Acad Sci USA 1995;92:9829–33
  • Boado RJ, Pardridge WM. The Trojan horse liposome technology for nonviral gene transfer across the blood–brain barrier. J Drug Deliv 2011;2011:296151
  • Swain GP, Prociuk M, Bagel JH, et al. Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain. Gene Ther 2014;21:28–36
  • Sondhi D, Johnson L, Purpura K, et al. Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis. Hum Gene Ther Methods 2012;23:324–35
  • Pardridge WM. Preparation of Trojan horse liposomes (THLs) for gene transfer across the blood–brain barrier. Cold Spring Harb Protoc 2010;2010:pdb prot5407
  • Nutt JG, Burchiel KJ, Comella CL, et al. Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD. Neurology 2003;60:69–73
  • Yan Q, Matheson C, Sun J, et al. Distribution of intracerebral ventricularly administered neurotrophins in rat brain and its correlation with trk receptor expression. Exp Neurol 1994;127:23–36
  • Lang AE, Gill S, Patel NK, et al. Randomized controlled trial of intraputamenal glial cell line-derived neurotrophic factor infusion in Parkinson disease. Ann Neurol 2006;59:459–66
  • Salvatore MF, Ai Y, Fischer B, et al. Point source concentration of GDNF may explain failure of phase II clinical trial. Exp Neurol 2006;202:497–505
  • Pardridge WM. Biopharmaceutical drug targeting to the brain. J Drug Target 2010;18:157–67
  • Pardridge WM. Gene targeting in vivo with pegylated immunoliposomes. Methods Enzymol 2003;373:507–28
  • Kissel K, Hamm S, Schulz M, et al. Immunohistochemical localization of the murine transferrin receptor (TfR) on blood–tissue barriers using a novel anti-TfR monoclonal antibody. Histochem Cell Biol 1998;110:63–72
  • Monnard PA, Oberholzer T, Luisi P. Entrapment of nucleic acids in liposomes. Biochim Biophys Acta 1997;1329:39–50
  • Lee HJ, Engelhardt B, Lesley J, et al. Targeting rat anti-mouse transferrin receptor monoclonal antibodies through blood–brain barrier in mouse. J Pharmacol Exp Ther 2000;292:1048–52
  • Zhang Y, Pardridge WM. Delivery of beta-galactosidase to mouse brain via the blood–brain barrier transferrin receptor. J Pharmacol Exp Ther 2005;313:1075–81
  • Shi N, Zhang Y, Zhu C, et al. Brain-specific expression of an exogenous gene after i.v. administration. Proc Natl Acad Sci USA 2001;98:12754–9
  • Bradbury MW. Transport of iron in the blood–brain–cerebrospinal fluid system. J Neurochem 1997;69:443–54
  • Moos T, Morgan EH. A morphological study of the developmentally regulated transport of iron into the brain. Dev Neurosci 2002;24:99–105
  • Ganesh S, Delgado-Escueta AV, Sakamoto T, et al. Targeted disruption of the Epm2a gene causes formation of Lafora inclusion bodies, neurodegeneration, ataxia, myoclonus epilepsy and impaired behavioral response in mice. Hum Mol Genet 2002;11:1251–62
  • Khatun R, Wu Y, Kanenishi K, et al. Immunohistochemical study of transferrin receptor expression in the placenta of pre-eclamptic pregnancy. Placenta 2003;24:870–6
  • Orberger G, Fuchs H, Geyer R, et al. Structural and functional stability of the mature transferrin receptor from human placenta. Arch Biochem Biophys 2001;386:79–88
  • Drake BL, Head JR. Transferrin receptor expression in early postimplantation mouse trophoblast and associated tissues. Placenta 1990;11:535–47
  • Takeda A, Takatsuka K, Connor JR, Oku N. Abnormal iron accumulation in the brain of neonatal hypotransferrinemic mice. Brain Res 2001;912:154–61
  • Zhang Y, Zhu C, Pardridge WM. Antisense gene therapy of brain cancer with an artificial virus gene delivery system. Mol Ther 2002;6:67–72
  • Chu C, Zhang Y, Boado RJ, Pardridge WM. Decline in exogenous gene expression in primate brain following intravenous administration is due to plasmid degradation. Pharm Res 2006;23:1586–90
  • Turnbull J, Girard JM, Lohi H, et al. Early-onset Lafora body disease. Brain 2012;135:2684–98
  • Ganesh S, Agarwala KL, Amano K, et al. Regional and developmental expression of Epm2a gene and its evolutionary conservation. Biochem Biophys Res Commun 2001;283:1046–53

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