Sendai virus-mediated CFTR gene transfer to the airway epithelium

Gene Therapy

Volumn 14, Issue 19, 2007, Pages 1371-1379

  Ferrari, S ^a,b   Griesenbach, U ^a,b   Iida, A ^c   Farley, R ^a,b   Wright, A M ^d   Zhu, J ^a   Munkonge, F M ^a,b   Smith, S N ^a,e   You, J ^c   Ban, H ^c   Inoue, M ^c   Chan, M ^a,b   Singh, C ^a,b   Verdon, B ^d   Argent, B E ^d   Wainwright, B ^f   Jeffery, P K ^a   Geddes, D M ^a,b   Porteous, D J ^b,e   Hyde, S C ^b,g   Gray, M A ^d   Hasegawa, M ^c   Alton, E W F W ^a,b   more..

^a IMPERIAL COLLEGE LONDON   (United Kingdom)

^b UK Cystic Fibrosis Gene Therapy Consortium ^*

^c University of Tokyo   (Japan)

^d NEWCASTLE UNIVERSITY   (United Kingdom)

^e UNIVERSITY OF EDINBURGH   (United Kingdom)

^f UNIVERSITY OF QUEENSLAND   (Australia)

^g UNIVERSITY OF OXFORD   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

CHLORIDE; COMPLEMENTARY DNA; TRANSMEMBRANE CONDUCTANCE REGULATOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL DIFFERENTIATION; CHLORIDE TRANSPORT; CONTROLLED STUDY; CYSTIC FIBROSIS; DNA SEQUENCE; ELECTRIC CURRENT; EPITHELIUM CELL; FEMALE; GENE DELETION; GENE INSERTION; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; IN VITRO STUDY; IN VIVO STUDY; INFLAMMATION; ION SELECTIVE ELECTRODE; MALE; MOUSE; MUTATIONAL ANALYSIS; NONHUMAN; NOSE MUCOSA; PATCH CLAMP; PRIORITY JOURNAL; RESPIRATORY EPITHELIUM; SENDAI VIRUS; VIRAL GENE DELIVERY SYSTEM; VIRUS RECOMBINANT;

AEROSOLS; ANIMALS; CHLORIDES; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; EPITHELIAL CELLS; FEMALE; GENE EXPRESSION; GENE THERAPY; GENETIC ENGINEERING; GENETIC VECTORS; IODIDES; ION CHANNELS; LUNG; MALE; MICE; MICE, KNOCKOUT; MUTATION; PATCH-CLAMP TECHNIQUES; SENDAI VIRUS; TRANSDUCTION, GENETIC;

MUS; SENDAI VIRUS;

EID: 34548730279     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/sj.gt.3302991     Document Type: Article

References (46)

1. Johnson LG, Olsen JC, Sarkadi B, Moore KL, Swanstrom R, Boucher RC. Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nat Genet 1992; 2: 21-25.
2. Dorin JR, Farley R, Webb S, Smith SN, Farini E, Delaney SJ et al. A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction. Gene Therapy 1996; 3: 797-801.
3. Ferrari S, Geddes DM, Alton EWFW. Barriers and new approaches for gene therapy and gene delivery in cystic fibrosis. Adv Drug Deliv Rev 2002; 54: 1373-1393.
4. Kitson C, Angel B, Judd D, Rothery S, Severs NJ, Dewar A et al. The extra- and intracellular barriers to lipid and adenovirus-mediated pulmonary gene transfer in native sheep airway epithelium. Gene Therapy 1999; 6: 534-546.
5. Ferrari S, Kitson C, Farley R, Steel R, Marriott C, Parkins DA et al. Mucus altering agents as adjuncts for nonviral gene transfer to airway epithelium. Gene Therapy 2001; 8: 1380-1386.
6. Wang G, Zabner J, Deering C, Launspach J, Shao J, Bodner M et al. Increasing epithelial junction permeability enhances gene transfer to airway epithelia in vivo. Am J Respir Cell Mol Biol 2000; 22 129-138.
7. Chu Q, St George JA, Lukason M, Cheng SH, Scheule RK, Eastman SJ. EGTA enhancement of adenovirus-mediated gene transfer to mouse tracheal epithelium in vivo. Hum Gene Ther 2001; 12: 455-467.
8. Duan D, Yue Y, Yan Z, Yang Y, Engelhardt JF. Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus. J Clin Invest 2000; 105: 1573-1587.
9. 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: 225-230.
10. Zhang L, Peeples ME, Boucher RC, Collins PL, Pickles RJ. Respiratory syncytial virus infection of human airway epithelial cells is polarized, specific to ciliated cells, and without obvious cytopathology. J Virol 2002; 76: 5654-5666.
11. Ziady AG, Kelley TJ, Milliken E, Ferkol T, Davis PB. Functional evidence of CFTR gene transfer in nasal epithelium of cystic fibrosis mice in vivo following luminal application of DANN complexes targeted to the serpin-enzyme complex receptor. Mol Ther 2002; 5: 413-419.
12. Yonemitsu Y, Kitson C, Ferrari S, Farley R, Griesenbach U, Judd D et al. Efficient gene transfer to airway epithelium using recombinant Sendai virus. Nat Biotechnol 2000; 18: 970-973.
13. Griesenbach U, Cassady RL, Ferrari S, Fukumura M, Muller C, Schmitt E et al. The nasal epithelium as a factory for systemic protein delivery. Mol Ther 2002; 5: 98-103.
14. Sheppard DN, Welsh MJ. Structure and function of the CFTR chloride channel. Physiol Rev 1999; 79 (1 Suppl): S23-S45.
15. Wilson R, Alton E, Rutman A, Higgins P, Al Nakib W, Geddes DM et al. Upper respiratory tract viral infection and mucociliary clearance. Eur J Respir Dis 1987; 70: 272-279.
16. Liang L, He C, Lei M, Li S, Hao Y, Zhu H et al. Pathology of guinea pigs experimentally infected with a novel reovirus and coronavirus isolated from SARS patients. DNA Cell Biol 2005; 24 485-490.
17. Chisari FV, Filippi P, Buras J, McLachlan A, Popper H, Pinkert CA et al. Structural and pathological effects of synthesis of hepatitis B virus large envelope polypeptide in transgenic mice. Proc Natl Acad Sci USA 1987; 84: 6909-6913.
18. Li HO, Zhu YF, Asakawa M, Kuma H, Hirata T, Ueda Y et al. A cytoplasmic RNA vector derived from nontransmissible Sendai virus with efficient gene transfer and expression. J Virol 2000; 74: 6564-6569.
19. Zhang L, Bukreyev A, Thompson CI, Watson B, Peeples ME, Collins PL et al. Infection of ciliated cells by human parainfluenza virus type 3 in an in vitro model of human airway epithelium. J Virol 2005; 79: 1113-1124.
20. Tabcharani JA, Linsdell P, Hanrahan JW. Halide permeation in wild type and mutant cystic fibrosis transmembrane conductance regulator chloride channels. J Gen Physiol 1997; 110: 341-354.
21. Gray MA, Harris A, Coleman L, Greenwell JR, Argent BE. Two types of chloride channel on duct cells cultured from human fetal pancreas. Am J Physiol 1989; 257: C240-C251.
22. - channels in CFTR-expressing Sf 9 cells switch spontaneously between slow and fast gating modes. Pflugers Arch - Eur J Physiol 1996; 432: 528-537.
23. Mohammad-Panah R, Demolombe S, Riochet D, Leblais V, Loussouran G, Pollard H et al. Hyperexpression of recombinant CFTR in heterologous cells alters its physiological properties. Am J Physiol 1998; 274: C310-C318.
24. Hurwitz JL, Soike KF, Sangster MY, Portner A, Sealy RE, Dawson DH et al. Intranasal Sendai virus vaccine protects African green monkeys from infection with human parainfluenza virus-type I. Vaccine 1997; 15: 533-540.
25. Slobod KS, Shenep JL, Lujan-Zilbermann J, Allison K, Brown B, Scroggs RA et al. Safety and immunogenicity of intranasal murine parainfluenza virus type 1 (Sendai virus) in healthy human adults. Vaccine 2004; 22: 3182-3186.
26. Engelhardt JF, Yankaskas JR, Ernst SA, Yang Y, Marino CR, Boucher RA et al. Submucosal glands are the predominant site of CFTR expression in human bronchus. Nat Genet 1992; 2: 240-247.
27. - transport and over-expression can generate basolateral CFTR. Am J Physiol Lung Cell Mol Physiol 2005; 289 L1123-1130.
28. Schiavi SC, Abdelkader N, Reber S, Pennington S, Narayana R, McPherson JM et al. Biosynthetic and growth abnormalities are associated with high-level expression of CFTR in heterologous cells. Am J Physiol Cell Physiol 1996; 270: C341-C351.
29. Ye L, Chan S, Chow YH, Tsui LC, Hu J. Regulated expression of the human CFTR gene in epithelial cells. Mol Ther 2001; 3: 723-733.
30. Tokusumi T, Iida A, Hirata T, Kato A, Nagai Y, Hasegawa M. Recombinant Sendai virus expressing different levels of a foreign gene. Virus Res 2002; 86: 33-38.
31. Inoue M, Tokusumi Y, Ban H, Shirakura M, Kanava T, Yoshizaki M et al. Recombinant Sendai virus vectors deleted in both the matrix and the fusion genes: Efficient gene transfer with preferable properties. J Gene Med 2004; 6: 1069-1081.
32. Yoshizaki M, Hironaka T, Iwasaki H, Ban H, Tokusumi Y, Iida A et al. Naked Sendai virus vector lacking all of the envelope-related genes: reduced cytopathogenicity and immunogenicity. J Gene Med 2006; 8: 1151-1159.
33. Griesenbach U, Mitomo K, Inoue M, Somerton L, Farley R, Singh C et al. Lentivirus pseudotyped with envelope proteins F and HN from Sendai virus transducer airway epithelium efficiently and persistently. Mol Ther 2006; 13 (Suppl 1) Abs 367: S139-S140.
34. Hasan MK, Kato A, Shioda T, Sakai Y, Yu D, Nagai Y. Creation of an infectious recombinant Sendai virus expressing the firefly luciferase gene from the 3′ proximal first locus. J Gen Virol 1997, 78 2813-2820.
35. Sakai Y, Kiyotani K, Fukumura. M, Asakawa M, Kato A, Shioda T et al. Accommodation of foreign genes into the Sendai virus genome: Sizes of inserted genes and viral replication. FEBS Lett 1999; 456: 221-226.
36. Kato A, Sakai Y, Shioda T, Kondo T, Nakanishi M, Nagai Y. Initiation of Sendai virus multiplication from transfected cDNA or RNA with negative or positive sense. Genes Cells 1996; 1: 569-579.
37. Yonemitsu Y, Kaneda Y. Hemagglutinating virus of Japan-liposome-mediated gene delivery to vascular cells. In Molecular Biology of Vascular Diseases. Method in Molecular Medicine. Human Press: Totowa, NJ, 1999, pp 295-306.
38. Denning GM, Anderson MP, Amara JF, Marshall J, Smith AE, Welsh MJ. Processing of mutant cystic fibrosis transmembrane conductance regulator is temperature-sensitive. Nature 1992; 358: 761-764.
39. -free medium. Biochem Biophys Res Commun 1996; 227: 755-761.
40. Wright AM, Gong X, Verdon B, Linsdell P, Mehta A, Riordan JR et al. Novel regulation of cystic fibrosis conductance regulator (CFTR) channel gating by external chloride. J Biol Chem 2004; 279: 41658-41663.
41. Venglarik CJ, Bridges RJ, Frizzell RA. A simple assay for agonist-regulated Cl and K conductances in salt-secreting epithelial cells. Am J Physiol 1990; 259: C358-C364.
42. - secretion in human squamous lung carcinoma epithelial cells. Biochim Biophys Acta 1994; 1224: 342-348.
43. Delaney SJ, Alton EWFW, Smith SN, Lunn DP, Farley R, Lovelock PK et al. Cystic fibrosis mice carrying the missense mutation G551D replicate human genotype-phenotype correlations. EMBO J 1996; 15: 955-963.
44. Zhou L, Dey CR, Wert SE, DuVall MD, Frizzell RA, Whitsett JA. Correction of lethal intestinal defect in a mouse model of cystic fibrosis by human CFTR. Science 1994; 266: 1705-1708.
45. Smith SN, Middleton PG, Chadwick S, Jaffe A, Bush KA, Rolleston S et al. The in vivo effects of Milrinone on the airways of cystic fibrosis mice and human subjects. Am J Respir Cell Mol Biol 1999; 20: 129-134.
46. Aherne WA, Dunnill MS. Point counting and morphometry. In: Aherne WA and Dunnill MS (eds). Morphometry. Arnold E: London, 1982, pp 33-45.