Advanced search
Publication Cover
Expert Opinion on Drug Delivery Volume 6, 2009 - Issue 6
Submit an article Journal homepage
442
Views
24
CrossRef citations to date
0
Altmetric
Reviews

Targeted gene delivery to the lung

Manish K Aneja Ludwig-Maximilians University, Division of Molecular Pulmonology, Department of Paediatrics, Lindwurmstrasse 2A, D-80337 Munich, Germany
, PhD,
Johannes-Peter Geiger Ludwig-Maximilians University, Division of Molecular Pulmonology, Department of Paediatrics, Lindwurmstrasse 2A, D-80337 Munich, Germany; Free University of Berlin, Department of Pharmacy, Takustrasse 3, 14166 Berlin, Germany +49 8951607711; +49 8951604421; [email protected]
,
Anne Himmel Ludwig-Maximilians University, Division of Molecular Pulmonology, Department of Paediatrics, Lindwurmstrasse 2A, D-80337 Munich, Germany; Free University of Berlin, Department of Pharmacy, Takustrasse 3, 14166 Berlin, Germany +49 8951607711; +49 8951604421; [email protected]
&
Carsten Rudolph Ludwig-Maximilians University, Division of Molecular Pulmonology, Department of Paediatrics, Lindwurmstrasse 2A, D-80337 Munich, Germany; Free University of Berlin, Department of Pharmacy, Takustrasse 3, 14166 Berlin, Germany +49 8951607711; +49 8951604421; [email protected]
, PhD
Pages 567-583 | Published online: 19 May 2009

Bibliography

  • Sanders N, Rudolph C, Braeckmans K, De Smedt SC, Demeester J. Extracellular barriers in respiratory gene therapy. Adv Drug Deliv Rev 2009;61(2):115-27
  • Davis PB, Cooper MJ. Vectors for airway gene delivery. AAPS J 2007;9(1):E11-17
  • Christian Plank FS, Carsten Rudolph: Localized Nucleic Acid Delivery: A Discussion of Selected Methods. In: Dr Martin S, editor, DNA-Pharmaceuticals, PlasmidFactory GmbH & Co. KG, Meisenstrasse 96, 33607 Bielefeld, Germany; 2006. p. 55-116
  • Yoshimura K, Rosenfeld MA, Nakamura H, et al. Expression of the human cystic fibrosis transmembrane conductance regulator gene in the mouse lung after in vivo intratracheal plasmid-mediated gene transfer. Nucleic Acids Res 1992;20(12):3233-40
  • Caplen NJ, Alton EW, Middleton PG, et al. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nat Med 1995;1(1):39-46
  • Ernst N, Ulrichskotter S, Schmalix WA, et al. Interaction of liposomal and polycationic transfection complexes with pulmonary surfactant. J Gene Med 1999;1(5):331-40
  • Gautam A, Densmore CL, Golunski E, et al. Transgene expression in mouse airway epithelium by aerosol gene therapy with PEI-DNA complexes. Mol Ther 2001;3(4):551-6
  • Bragonzi A, Conese M. Non-viral approach toward gene therapy of cystic fibrosis lung disease. Curr Gene Ther 2002;2(3):295-305
  • Rudolph C, Lausier J, Naundorf S, et al. In vivo gene delivery to the lung using polyethylenimine and fractured polyamidoamine dendrimers. J Gene Med 2000;2(4):269-78
  • Ferrari S, Geddes DM, Alton EW. Barriers to and new approaches for gene therapy and gene delivery in cystic fibrosis. Adv Drug Deliv Rev 2002;54(11):1373-93
  • Zabner J, Cheng SH, Meeker D, et al. Comparison of DNA-lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelia in vivo. J Clin Invest 1997;100(6):1529-37
  • 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(2):1117-26
  • Jiang C, O'Connor SP, Fang SL, et al. Efficiency of cationic lipid-mediated transfection of polarized and differentiated airway epithelial cells in vitro and in vivo. Hum Gene Ther 1998;9(11):1531-42
  • Kitson C, Angel B, Judd D, et al. The extra- and intracellular barriers to lipid and adenovirus-mediated pulmonary gene transfer in native sheep airway epithelium. Gene Ther 1999;6(4):534-46
  • Coyne CB, Kelly MM, Boucher RC, Johnson LG. Enhanced epithelial gene transfer by modulation of tight junctions with sodium caprate. Am J Respir Cell Mol Biol 2000;23(5):602-9
  • Seiler MP, Luner P, Moninger TO, et al. Thixotropic solutions enhance viral-mediated gene transfer to airway epithelia. Am J Respir Cell Mol Biol 2002;27(2):133-40
  • Ferrari S, Kitson C, Farley R, et al. Mucus altering agents as adjuncts for nonviral gene transfer to airway epithelium. Gene Ther 2001;8(18):1380-6
  • Sanders NN, Van Rompaey E, De Smedt SC, Demeester J. Structural alterations of gene complexes by cystic fibrosis sputum. Am J Respir Crit Care Med 2001;164(3):486-93
  • Rosenecker J, Naundorf S, Gersting SW, et al. Interaction of bronchoalveolar lavage fluid with polyplexes and lipoplexes: analysing the role of proteins and glycoproteins. J Gene Med 2003;5(1):49-60
  • Pickles RJ, McCarty D, Matsui H, et al. Limited entry of adenovirus vectors into well-differentiated airway epithelium is responsible for inefficient gene transfer. J Virol 1998;72(7):6014-23
  • Scherer F, Anton M, Schillinger U, et al. Magnetofection: enhancing and targeting gene delivery by magnetic force in vitro and in vivo. Gene Ther 2002;9(2):102-9
  • Plank C, Schillinger U, Scherer F, et al. The magnetofection method: using magnetic force to enhance gene delivery. Biol Chem 2003;384(5):737-47
  • Gersting SW, Schillinger U, Lausier J, et al. Gene delivery to respiratory epithelial cells by magnetofection. J Gene Med 2004;6(8):913-22
  • Xenariou S, Griesenbach U, Ferrari S, et al. Using magnetic forces to enhance non-viral gene transfer to airway epithelium in vivo. Gene Ther 2006;13(21):1545-52
  • Schillinger U, Brill T, Rudolph C, et al. Advances in magnetofection–magnetically guided nucleic acid delivery. Proceedings of the Fifth International Conference on Scientific and Clinical Apllications of Magnetic Carriers. J Magnetism Magn Mater 2005;293(1):501-8
  • Mykhaylyk O, Antequera YS, Vlaskou D, Plank C. Generation of magnetic nonviral gene transfer agents and magnetofection in vitro. Nat Protoc 2007;2(10):2391-411
  • Dames P, Gleich B, Flemmer A, et al. Targeted delivery of magnetic aerosol droplets to the lung. Nat Nanotechnol 2007;2(8):495-9
  • Lavigne MD, Gorecki DC. Emerging vectors and targeting methods for nonviral gene therapy. Expert Opin Emerg Drugs 2006;11(3):541-57
  • Mehier-Humbert S, Guy RH. Physical methods for gene transfer: improving the kinetics of gene delivery into cells. Adv Drug Deliv Rev 2005;57(5):733-53
  • Gehl J. Electroporation: theory and methods, perspectives for drug delivery, gene therapy and research. Acta Physiol Scand 2003;177(4):437-47
  • Wells DJ. Gene therapy progress and prospects: electroporation and other physical methods. Gene Ther 2004;11(18):1363-9
  • Neumann E, Schaefer-Ridder M, Wang Y, Hofschneider PH. Gene transfer into mouse lyoma cells by electroporation in high electric fields. EMBO J 1982;1(7):841-5
  • Titomirov AV, Sukharev S, Kistanova E. In vivo electroporation and stable transformation of skin cells of newborn mice by plasmid DNA. Biochim Biophys Acta 1991;1088(1):131-4
  • Aihara H, Miyazaki J-i. Gene transfer into muscle by electroporation in vivo. Nat Biotech 1998;16(9):867-70
  • Vanbever R, Préat V. In vivo efficacy and safety of skin electroporation. Adv Drug Deliv Rev 1999;35(1):77-88
  • Heller R, Jaroszeski M, Atkin A, et al. In vivo gene electroinjection and expression in rat liver. FEBS Lett 1996;389(3):225-8
  • Dean DA, Machado-Aranda D, Blair-Parks K, et al. Electroporation as a method for high-level nonviral gene transfer to the lung. Gene Ther 2000;10(18):1608-15
  • Zhang Q, Wang Z, Ran H, et al. Enhanced gene delivery into skeletal muscles with ultrasound and microbubble techniques. Acad Radiol 2006;13(3):363-7
  • Lu QL, Liang HD, Partridge T, Blomley MJK. Microbubble ultrasound improves the efficiency of gene transduction in skeletal muscle in vivo with reduced tissue damage. Gene Ther 2003;10(5):396-405
  • Miller DL, Bao S, Gies RA, Thrall BD. Ultrasonic enhancement of gene transfection in murine melanoma tumors. Ultrasound Med Biol 1999;25(9):1425-30
  • Miller DL, Song J. Tumor growth reduction and DNA transfer by cavitation-enhanced high-intensity focused ultrasound in vivo. Ultrasound Med Biol 2003;29(6):887-93
  • Lubbe AS, Bergemann C, Riess H, et al. Clinical experiences with magnetic drug targeting: a phase I study with 4′-epidoxorubicin in 14 patients with advanced solid tumors. Cancer Res 1996;56(20):4686-93
  • Dean DA. Electroporation of the vasculature and the lung. DNA Cell Biol 2003;22(12):797-806
  • Anwer K, Kao G, Proctor B, et al. Ultrasound enhancement of cationic lipid-mediated gene transfer to primary tumors following systemic administration. Gene Therapy 2000;21:1833-9
  • Lawrie A, Brisken AF, Francis SE, et al. Microbubble-enhanced ultrasound for vascular gene delivery. Gene Ther 2000;7(23):2023-7
  • Brown MD, Schatzlein AG, Uchegbu IF. Gene delivery with synthetic (non viral) carriers. Int J Pharm 2001;229(1-2):1-21
  • Lee TWR, Matthews DA, Blair GE. Novel molecular approaches to cystic fibrosis gene therapy. Biochem J 2005;387:1-15
  • Guillaume-Gable C, Floch V, Mercier B, et al. Cationic phosphonolipids as nonviral gene transfer agents in the lungs of mice. Hum Gene Ther 1998;9(16):2309-19
  • Barron LG, Gagne L, Szoka FC Jr. Lipoplex-mediated gene delivery to the lung occurs within 60 minutes of intravenous administration. Hum Gene Ther 1999;10(10):1683-94
  • Ruiz FE, Clancy JP, Perricone MA, et al. A clinical inflammatory syndrome attributable to aerosolized lipid-DNA administration in cystic fibrosis. Hum Gene Ther 2001;12(7):751-61
  • Parker AL, Newman C, Briggs S, et al. Nonviral gene delivery: techniques and implications for molecular medicine. Expert Rev Mol Med 2003;5(22):1-15
  • De Smedt SC, Demeester J, Hennink WE. Cationic polymer based gene delivery systems. Pharm Res 2000;17(2):113-26
  • Han S, Mahato RI, Sung YK, Kim SW. Development of biomaterials for gene therapy. Mol Ther 2000;2(4):302-17
  • Zou SM, Erbacher P, Remy JS, Behr JP. Systemic linear polyethylenimine (L-PEI)-mediated gene delivery in the mouse. J Gene Med 2000;2(2):128-34
  • Densmore CL, Orson FM, Xu B, et al. Aerosol delivery of robust polyethyleneimine-DNA complexes for gene therapy and genetic immunization. Mol Ther 2000;1(2):180-8
  • Rudolph C, Schillinger U, Ortiz A, et al. Aerosolized nanogram quantities of plasmid DNA mediate highly efficient gene delivery to mouse airway epithelium. Mol Ther 2005;12(3):493-501
  • Davies LA, Seguela C, Varathalingam A, Cheng SH, Hyde SC, Gill DR. Identification of transfected cell types following non-viral gene transfer to the murine lung. J Gene Med 2007;9(3):184-96
  • Weiss SI, Sieverling N, Niclasen M, et al. Uronic acids functionalized polyethyleneimine (PEI)-polyethyleneglycol (PEG)-graft-copolymers as novel synthetic gene carriers. Biomaterials 2006;27(10):2302-12
  • Elfinger M, Maucksch C, Rudolph C. Characterization of lactoferrin as a targeting ligand for nonviral gene delivery to airway epithelial cells. Biomaterials 2007;28(23):3448-55
  • Ziady AG, Kim J, Colla J, Davis PB. Defining strategies to extend duration of gene expression from targeted compacted DNA vectors. Gene Ther 2004;11(18):1378-90
  • Elfinger M, Geiger J, Hasenpusch G, et al. Targeting of the β2-adrenoceptor increases nonviral gene delivery to pulmonary epithelial cells in vitro and lungs in vivo. J Control Release 2009;135(3):234-41
  • Ziady AG, Kelley TJ, Milliken E, et al. Functional evidence of CFTR gene transfer in nasal epithelium of cystic fibrosis mice in vivo following luminal application of DNA complexes targeted to the serpin-enzyme complex receptor. Mol Ther 2002;5(4):413-9
  • Oudrhiri N, Vigneron JP, Peuchmaur M, et al. Gene transfer by guanidinium-cholesterol cationic lipids into airway epithelial cells in vitro and in vivo. Proc Natl Acad Sci USA 1997;94(5):1651-6
  • Li S, Huang L. In vivo gene transfer via intravenous administration of cationic lipid-protamine-DNA (LPD) complexes. Gene Ther 1997;4(9):891-900
  • Noone PG, Hohneker KW, Zhou Z, et al. Safety and biological efficacy of a lipid-CFTR complex for gene transfer in the nasal epithelium of adult patients with cystic fibrosis. Mol Ther 2000;1(1):105-14
  • Urtti A, Polansky J, Lui GM, Szoka FC. Gene delivery and expression in human retinal pigment epithelial cells: effects of synthetic carriers, serum, extracellular matrix and viral promoters. J Drug Target 2000;7(6):413-21
  • Zelphati O, Nguyen C, Ferrari M, et al. Stable and monodisperse lipoplex formulations for gene delivery. Gene Ther 1998;5(9):1272-82
  • Stavridis JC, Deliconstantinos G, Psallidopoulos MC, et al. Construction of transferrin-coated liposomes for in vivo transport of exogenous DNA to bone marrow erythroblasts in rabbits. Exp Cell Res 1986;164(2):568-72
  • Dzau VJ, Mann MJ, Morishita R, Kaneda Y. Fusigenic viral liposome for gene therapy in cardiovascular diseases. Proc Natl Acad Sci USA 1996;93(21):11421-5
  • Willis M, Forssen E. Ligand-targeted liposomes. Adv Drug Deliv Rev 1998;29(3):249-71
  • Cryan SA. Carrier-based strategies for targeting protein and peptide drugs to the lungs. AAPS J 2005;7(1):E20-41
  • Tagalakis AD, McAnulty RJ, Devaney J, et al. A receptor-targeted nanocomplex vector system optimized for respiratory gene transfer. Mol Ther 2008;16(5):907-15
  • Wattiaux R, Laurent N, Wattiaux-De Coninck S, Jadot M. Endosomes, lysosomes: their implication in gene transfer. Adv Drug Deliv Rev 2000;41(2):201-8
  • Mastrobattista E, Koning GA, van Bloois L, et al. Functional characterization of an endosome-disruptive peptide and its application in cytosolic delivery of immunoliposome-entrapped proteins. J Biol Chem 2002;277(30):27135-43
  • Boussif O, Lezoualc'h F, Zanta MA, et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc Natl Acad Sci USA 1995;92(16):7297-301
  • Akinc A, Thomas M, Klibanov AM, Langer R. Exploring polyethylenimine-mediated DNA transfection and the proton sponge hypothesis. J Gene Med 2005;7(5):657-63
  • Lukacs GL, Haggie P, Seksek O, et al. Size-dependent DNA mobility in cytoplasm and nucleus. J Biol Chem 2000;275(3):1625-9
  • Suh J, Wirtz D, Hanes J. Real-time intracellular transport of gene nanocarriers studied by multiple particle tracking. Biotechnol Prog 2004;20(2):598-602
  • Ludtke JJ, Zhang GF, Sebestyen MG, Wolff JA. A nuclear localization signal can enhance both the nuclear transport and expression of 1 kb DNA. J Cell Sci 1999;112(12):2033-41
  • Chen X, Kube DM, Cooper MJ, Davis PB. Cell surface nucleolin serves as receptor for DNA nanoparticles composed of pegylated polylysine and DNA. Mol Ther 2008;16(2):333-42
  • Chan CK, Senden T, Jans DA. Supramolecular structure and nuclear targeting efficiency determine the enhancement of transfection by modified polylysines. Gene Ther 2000;7(19):1690-7
  • Sandgren S, Cheng F, Belting M. Nuclear targeting of macromolecular polyanions by an HIV-Tat derived peptide - Role for cell-surface proteoglycans. J Biol Chem 2002;277(41):38877-83
  • Rudolph C, Plank C, Lausier J, et al. Oligomers of the arginine-rich motif of the HIV-1 TAT protein are capable of transferring plasmid DNA into cells. J Biol Chem 2003;278(13):11411-8
  • Klink DT, Chao S, Glick MC, Scanlin TF. Nuclear translocation of lactosylated poly-L-lysine/cDNA complex in cystic fibrosis airway epithelial cells. Mol Ther 2001;3(6):831-41
  • Summerford C, Samulski RJ. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 1998;72(2):1438-45
  • Goldman MJ, Lee PS, Yang JS, Wilson JM. Lentiviral vectors for gene therapy of cystic fibrosis. Hum Gene Ther 1997;8(18):2261-8
  • Wang G, Slepushkin V, Zabner J, et al. Feline immunodeficiency virus vectors persistently transduce nondividing airway epithelia and correct the cystic fibrosis defect. J Clin Invest 1999;104(11):R55-62
  • Kobayashi M, Iida A, Ueda Y, Hasegawa M. Pseudotyped lentivirus vectors derived from simian immunodeficiency virus SIVagm with envelope glycoproteins from paramyxovirus. J Virol 2003;77(4):2607-14
  • Tarantal AF, Lee CI, Ekert JE, et al. Lentiviral vector gene transfer into fetal rhesus monkeys (Macaca mulatta): lung-targeting approaches. Mol Ther 2001;4(6):614-21
  • Tarantal AF, McDonald RJ, Jimenez DF, et al. Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery. Mol Ther 2005;12(1):87-98
  • Lim FY, Kobinger GP, Weiner DJ, et al. Human fetal trachea-scid mouse xenografts: Efficacy of vesicular stomatitis virus-G pseudotyped lentiviral-mediated gene transfer. J Pediatric Surg 2003;38(6):834-9
  • Johnson LG, Olsen JC, Naldini L, Boucher RC. Pseudotyped human lentiviral vector-mediated gene transfer to airway epithelia in vivo. Gene Ther 2000;7(7):568-74
  • Limberis M, Anson DS, Fuller M, Parsons DW. Recovery of airway cystic fibrosis transmembrane conductance regulator function in mice with cystic fibrosis after single-dose lentivirus-mediated gene transfer. Hum Gene Ther 2002;13(16):1961-70
  • Medina MF, Kobinger GP, Rux J, et al. Lentiviral vectors pseudotyped with minimal filovirus envelopes increased gene transfer in murine lung. Mol Ther 2003;8(5):777-89
  • Sinn PL, Hickey MA, Staber PD, et al. Lentivirus vectors pseudotyped with filoviral envelope glycoproteins transduce airway epithelia from the apical surface independently of folate receptor alpha. J Virol 2003;77(10):5902-10
  • Sinn PL, Burnight ER, Hickey MA, et al. Persistent gene expression in mouse nasal epithelia following feline immunodeficiency virus-based vector gene transfer. J Virol 2005;79(20):12818-27
  • McKay T, Patel M, Pickles RJ, et al. Influenza M2 envelope protein augments avian influenza hemagglutinin pseudotyping of lentiviral vectors. Gene Ther 2006;13(8):715-24
  • Kremer KL, Dunning KR, Parsons DW, Anson DS. Gene delivery to airway epithelial cells in vivo: a direct comparison of apical and basolateral transduction strategies using pseudotyped lentivirus vectors. J Gene Med 2007;9(5):362-8
  • Kobinger GP, Weiner DJ, Yu QC, Wilson JM. Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo. Nat Biotechnol 2001;19(3):225-30
  • Morizono K, Chen ISY. Targeted gene delivery by intravenous injection of retroviral vectors. Cell Cycle 2005;4(7):854-6
  • Morizono K, Xie YM, Ringpis GE, et al. Lentiviral vector retargeting to P-glycoprotein on metastatic melanoma through intravenous injection. Nat Med 2005;11(3):346-52
  • Sinn PL, Arias AC, Brogden KA, McCray PB Jr. Lentivirus vector can be readministered to nasal epithelia without blocking immune responses. J Virol 2008;82(21):10684-92
  • Hacein-Bey-Abina S, Von Kalle C, Schmidt M, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003;302(5644):415-9
  • Bartholomae CC, Yanez-Munoz RJ, Balaggan KS, et al. Nearly random integration site distribution in postmitotic rodent tissues transduced with lentiviral SIN-vectors. Blood Cells Mol Dis 2008;40(2):253
  • St George JA. Gene therapy progress and prospects: adenoviral vectors. Gene Ther 2003;10(14):1135-41
  • Cao HB, Koehler DR, Hu J. Adenoviral vectors for gene replacement therapy. Viral Immunol 2004;17(3):327-33
  • Grubb BR, Pickles RJ, Ye H, et al. Inefficient gene-transfer by adenovirus vector to cystic-fibrosis airway epithelia of mice and humans. Nature 1994;371(6500):802-6
  • Harvey BG, Leopold PL, Hackett NR, et al. Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirus. J Clin Invest 1999;104(9):1245-55
  • Joseph PM, O'Sullivan B, Lapey A, et al. Aerosol and lobar administration of a recombinant adenovirus to individuals with cystic fibrosis. I. Methods, safety, and clinical implications. Hum Gene Ther 2001;12(11):1369-82
  • Perricone MA, Morris JE, Pavelka K, et al. Aerosol and lobar administration of a recombinant adenovirus to individuals with cystic fibrosis. II. Transfection efficiency in airway epithelium. Hum Gene Ther 2001;12(11):1383-94
  • Zuckerman JB, Robinson CB, McCoy KS, et al. A phase I study of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator gene to a lung segment of individuals with cystic fibrosis. Hum Gene Ther 1999;10(18):2973-85
  • Pickles RJ, Fahrner JA, Petrella JM, et al. Retargeting the coxsackievirus and adenovirus receptor to the apical surface of polarized epithelial cells reveals the glycocalyx as a barrier to adenovirus-mediated gene transfer. J Virol 2000;74(13):6050-7
  • Lee JH, Zabner J, Welsh MJ. Delivery of an adenovirus vector in a calcium phosphate coprecipitate enhances the therapeutic index of gene transfer to airway epithelia. Hum Gene Ther 1999;10(4):603-13
  • Chu Q, St George JA, Lukason M, et al. EGTA enhancement of adenovirus-mediated gene transfer to mouse tracheal epithelium in vivo. Hum Gene Ther 2001;12(5):455-67
  • Wang GS, Zabner J, Deering C, et al. Increasing epithelial junction permeability enhances gene transfer to airway epithelia in vivo. Am J Respir Cell Mol Biol 2000;22(2):129-38
  • Kaplan JM, Pennington SE, St George JA, et al. Potentiation of gene transfer to the mouse lung by complexes of adenovirus vector and polycations improves therapeutic potential. Hum Gene Ther 1998;9(10):1469-79
  • Parsons DW, Grubb BR, Johnson LG, Boucher RC. Enhanced in vivo airway gene transfer via transient modification of host barrier properties with a surface-active agent. Hum Gene Ther 1998;9(18):2661-72
  • Johnson LG, Vanhook MK, Coyne CB, et al. Safety and efficiency of modulating paracellular permeability to enhance airway epithelial gene transfer in vivo. Hum Gene Ther 2003;14(8):729-47
  • Koehler DR, Frndova H, Leung K, et al. Aerosol delivery of an enhanced helper-dependent adenovirus formulation to rabbit lung using an intratracheal catheter. J Gene Med 2005;7(11):1409-20
  • Yei SP, Mittereder N, Wert S, et al. In-vivo evaluation of the safety of adenovirus-mediated transfer of the human cystic-fibrosis transmembrane conductance regulator Cdna to the lung. Hum Gene Ther 1994;5(6):731-44
  • Wilmott RW, Amin RS, Perez CR, et al. Safety of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator cDNA to the lungs of nonhuman primates. Hum Gene Ther 1996;7(3):301-18
  • Simon RH, Engelhardt JF, Yang YP, et al. Adenovirus-Mediated Transfer of the Cftr Gene to Lung of Nonhuman-Primates – Toxicity Study. Hum Gene Ther 1993;4(6):771-80
  • Harvey BG, Maroni J, O'Donoghue KA, et al. Safety of local delivery of low- and intermediate-dose adenovirus gene transfer vectors to individuals with a spectrum of morbid conditions. Hum Gene Ther 2002;13(1):15-63
  • Dai Y, Schwarz EM, Gu D, et al. Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression. Proc Natl Acad Sci USA 1995;92(5):1401-5
  • Morral N, O'Neal W, Zhou H, et al. Immune responses to reporter proteins and high viral dose limit duration of expression with adenoviral vectors: comparison of E2a wild type and E2a deleted vectors. Hum Gene Ther 1997;8(10):1275-86
  • O'Neal WK, Zhou H, Morral N, et al. Toxicological comparison of E2a-deleted and first-generation adenoviral vectors expressing alpha1-antitrypsin after systemic delivery. Hum Gene Ther 1998;9(11):1587-98
  • Yang Y, Nunes FA, Berencsi K, et al. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc Natl Acad Sci USA 1994;91(10):4407-11
  • Yang Y, Li Q, Ertl HC, Wilson JM. Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J Virol 1995;69(4):2004-15
  • Chirmule N, Hughes JV, Gao GP, et al. Role of E4 in eliciting CD4 T-cell and B-cell responses to adenovirus vectors delivered to murine and nonhuman primate lungs. J Virol 1998;72(7):6138-45
  • Engelhardt JF, Litzky L, Wilson JM. Prolonged transgene expression in cotton rat lung with recombinant adenoviruses defective in E2a. Hum Gene Ther 1994;5(10):1217-29
  • Goldman MJ, Litzky LA, Engelhardt JF, Wilson JM. Transfer of the CFTR gene to the lung of nonhuman primates with E1-deleted, E2a-defective recombinant adenoviruses: a preclinical toxicology study. Hum Gene Ther 1995;6(7):839-51
  • Walters RW, Grunst T, Bergelson JM, et al. Basolateral localization of fiber receptors limits adenovirus infection from the apical surface of airway epithelia. J Biol Chem 1999;274(15):10219-26
  • Palmer DJ, Ng P. Helper-dependent adenoviral vectors for gene therapy. Hum Gene Ther 2005;16(1):1-16
  • Hamilton H, Gomos J, Berns KI, Falck-Pedersen E. Adeno-associated virus site-specific integration and AAVS1 disruption. J Virol 2004;78(15):7874-82
  • Flotte TR. Adeno-associated virus-mediated gene transfer for lung diseases. Hum Gene Ther 2005;16(6):643-8
  • Halbert CL, Allen JM, Miller AD. Adeno-associated virus type 6 (AAV6) vectors mediate efficient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectors. J Virol 2001;75(14):6615-24
  • Halbert CL, Rutledge EA, Allen JM, et al. Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes. J Virol 2000;74(3):1524-32
  • Limberis MP, Wilson JM. Adeno-associated virus serotype 9 vectors transduce murine alveolar and nasal epithelia and can be readministered. Proc Natl Acad Sci USA 2006;103(35):12993-8
  • Seiler MP, Miller AD, Zabner J, Halbert CL. Adeno-associated virus types 5 and 6 use distinct receptors for cell entry. Hum Gene Ther 2006;17(1):10-9
  • Sumner-Jones SG, Gill DR, Hyde SC. Lack of repeat transduction by recombinant adeno-associated virus type 5/5 vectors in the mouse airway. J Virol 2007;81(22):12360-7
  • Halbert CL, Miller AD, McNamara S, et al. Prevalence of neutralizing antibodies against adeno-associated virus (AAV) types 2, 5, and 6 in cystic fibrosis and normal populations: implications for gene therapy using AAV vectors. Hum Gene Ther 2006;17(4):440-7
  • Li S, Tan Y, Viroonchatapan E, et al. Targeted gene delivery to pulmonary endothelium by anti-PECAM antibody. Am J Physiol Lung Cell Mol Physiol 2000;278(3):L504-511
  • Cristiano RJ, Roth JA. Epidermal growth factor mediated DNA delivery into lung cancer cells via the epidermal growth factor receptor. Cancer Gene Ther 1996;3(1):4-10
  • Song S, Liu D, Peng J, et al. Peptide ligand-mediated liposome distribution and targeting to EGFR expressing tumor in vivo. Int J Pharm 2008;363(1-2):155-61
  • Kollen WJ, Midoux P, Erbacher P, et al. Gluconoylated and glycosylated polylysines as vectors for gene transfer into cystic fibrosis airway epithelial cells. Hum Gene Ther 1996;7(13):1577-86
  • Grosse S, Thévenot G, Aron Y, et al. In vivo gene delivery in the mouse lung with lactosylated polyethylenimine, questioning the relevance of in vitro experiments. J Control Release 2008;132(2):105-12
  • Jost PJ, Harbottle RP, Knight A, et al. A novel peptide, THALWHT, for the targeting of human airway epithelia. FEBS Lett 2001;489(2-3):263-9
  • Jenkins RG, Herrick SE, Meng QH, et al. An integrin-targeted non-viral vector for pulmonary gene therapy. Gene Ther 2000;7(5):393-400
  • Chang DK, Lin CT, Wu CH, Wu HC. A novel peptide enhances therapeutic efficacy of liposomal anti-cancer drugs in mice models of human lung cancer. PLoS ONE 2009;4(1):e4171
  • Liu X, Luo M, Guo C, et al. Analysis of adeno-associated virus progenitor cell transduction in mouse lung. Mol Ther 2008;17(2):285-93
  • Halbert CL, Lam SL, Miller AD. High-efficiency promoter-dependent transduction by adeno-associated virus type 6 vectors in mouse lung. Hum Gene Ther 2007;18(4):344-54
  • Limberis MP, Vandenberghe LH, Zhang L, et al. Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro. Mol Ther 2009;17(2):294-301
  • Gilbert JL, Purcell J, Strappe P, et al. Comparative evaluation of viral, nonviral and physical methods of gene delivery to normal and transformed lung epithelial cells. Anticancer Drugs 2008;19(8):783-8
  • Buckley SM, Howe SJ, Sheard V, et al. Lentiviral transduction of the murine lung provides efficient pseudotype and developmental stage-dependent cell-specific transgene expression. Gene Ther 2008;15(16):1167-75
  • Mitomo K, Inoue M, Ueda Y, et al. 355. Towards cystic fibrosis and airway gene therapy: evaluation of EGFP gene expression in murine nasal airways mediated by simian immunodeficiency virus vectors pseudotyped with sendai virus glycoproteins F and HN. Mol Ther 2005;11(S1):S139-S139
  • Shirohzu H, Mitomo K, Tabata T, et al. 495. Efficient in vivo transduction of mouse airway epithelial cells by the simian immunodeficiency virus vector pseudotyped with sendai virus F and HN proteins. Mol Ther 2004;9(S1):S188-S188
  • Kobinger GP, Limberis MP, Somanathan S, et al. Human immunodeficiency viral vector pseudotyped with the spike envelope of severe acute respiratory syndrome coronavirus transduces human airway epithelial cells and dendritic cells. Hum Gene Ther 2007;18(5):413-22
  • Katkin JP, Gilbert BE, Langston C, et al. Aerosol delivery of a beta-galactosidase adenoviral vector to the lungs of rodents. Hum Gene Ther 1995;6(8):985-95
  • Conzelmann KK. Nonsegmented negative-strand RNA viruses: genetics and manipulation of viral genomes. Annu Rev Genet 1998;32:123-62
  • Ferrari S, Griesenbach U, Iida A, et al. Sendai virus-mediated CFTR gene transfer to the airway epithelium. Gene Ther 2007;14(19):1371-9

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Order Reprints Request Corporate Permissions

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

Request Academic Permissions

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.

Related research

People also read lists articles that other readers of this article have read.

Recommended articles lists articles that we recommend and is powered by our AI driven recommendation engine.

Cited by lists all citing articles based on Crossref citations.
Articles with the Crossref icon will open in a new tab.