Enhanced in vivo airway gene transfer via transient modification of host barrier properties with a surface-active agent

Human Gene Therapy

Volumn 9, Issue 18, 1998, Pages 2661-2672

  Parsons, David W ^a,b   Grubb, Barbara R ^a   Johnson, Larry G ^a   Boucher, Richard C ^a  

^a UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

^b WOMEN S AND CHILDREN S HOSPITAL   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

DETERGENT; LANTHANUM; POLIDOCANOL; TRANSMEMBRANE CONDUCTANCE REGULATOR;

ADENOVIRUS; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CELL JUNCTION; CELL MEMBRANE PERMEABILITY; CONTROLLED STUDY; FEMALE; GENE EXPRESSION; GENE TRANSFER; MOUSE; NONHUMAN; NOSE INFECTION; NOSE MUCOSA;

ADENOVIRIDAE; ANIMALIA; MURINAE;

EID: 0032506749     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1998.9.18-2661     Document Type: Article

References (46)

1. BAGLIONI, C., and PHIPPS, R.J. (1990). Nasal absorption of interferon: enhancement by surfactant agents. J. Interferon Res. 10, 497-504.
2. BOUCHER, R.C. (1996). Current status of CF gene therapy. Trends Genet. 12, 81-84.
3. CARSTENS, S., DANIELSEN, G., GULDHAMMER, B., and FREDERIKSEN, O. (1993). Transport of insulin across rabbit nasal mucosa in vitro induced by didecanoyl-L-α-phosphatidylcholine. Diabetes 42, 1032-1040.
4. CLARKE, L.L., GRUBB, B.R., GABRIEL, S.E., SMITHIES, O., KOLLER, B.H., and BOUCHER, R.C. (1992). Defective epithelial chloride transport in a gene targeted mouse model of cystic fibrosis. Science 257, 1125-1128.
5. CRYSTAL, R.G., McELVANEY, N.G., ROSENFELD, M.A., CHU, C.S., MASTRANGELI, A., HAY, J.G., BRODY, S.L., JAFFE, H.A., EISSA, N.T., and DANEL, C. (1994). Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nature Genet. 8, 42-51. [See comments]
6. CRYSTAL, R.G., JAFFE, A., BRODY, S., MASTRANGELI, A., McELVANEY, N.G., ROSENFELD, M., CHU, C.S., DANEL, C., HAY, J., and EISSA, T. (1995). A phase 1 study, in cystic fibrosis patients, of the safety, toxicity, and biological efficacy of a single administration of a replication deficient, recombinant adenovirus carrying the cDNA of the normal cystic fibrosis transmembrane conductance regulator gene in the lung. Hum. Gene Ther. 6, 643-666.
7. DAUGHERTY, A.L., LIGGITT, H.D., McCABE, J.G., MOORE, J.A., and PATTON, J.S. (1988). Absorption of recombinant methionyl-human growth hormone (Met-hGH) from rat nasal mucosa. Int. J. Pharm. 45, 197-206.
8. DONG, J.Y., WANG, D., VAN GINKEL, F.W., PASCUAL, D.W., and FRIZZELL, R.A. (1996). Systematic analysis of repeated gene delivery into animal lungs with a recombinant adenovirus vector. Hum. Gene Ther. 7, 319-331.
9. DORIN, J.R., FARLEY, R., WEBB, S., SMITH, S.N., FARINI, E., DELANEY, S.J., WAINWRIGHT, B.J., ALTON, E.W., and PORTEOUS, D.J. (1996). A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction. Gene Ther. 3, 797-801.
10. DRUMM, M.L., POPE, H.A., CLIFF, W.H., ROMMENS, J.M., MARVIN, S.A., TSUI, L.C., COLLINS, F.S., FRIZZELL, R.A., and WILSON, J.M. (1990). Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer. Cell 62, 1227-1233. [Published erratum appears in Cell 1993 Jun 16;74(1):215].
11. DUPUIT, F., ZAHM, J.M., PIERROT, D., BREZILLON, S., BONNET, N., IMLER, J.L., PAVIRANI, A., and PUCHELLE, E. (1995). Regenerating cells in human airway surface epithelium represent preferential targets for recombinant adenovirus. Hum. Gene Ther. 6, 1185-1193.
12. ENGELHARDT, J.F., STRATFORD-PERRICAUDET, L.D., YANG, Y., KAZARSKI, K., PERRICAUDET, M., YANKASKAS, J.R., and WILSON, J.M. (1993). Direct transfer of recombinant genes into human bronchial epithelia of xenografts with E1 deleted adenoviruses. Nature Genet. 4, 27-34.
13. ESKENASY, A. (1980). Bronchiolar pathology II. Light and electron microscopical changes of bronchiolar epithelial cells in rabbits repeatedly injected intratracheally with a detergent solution. Revue roumaine de morphologie, d'embryologie et de physiologie Morphologie-embryologie. Morphologie et embryologie. Morphol Embryol 26, 279-284.
14. GOLDMAN, P.M. (1989). Polidocanol (Aethoxysklerol) for sclerotherapy of superficial venules and telangiectasias. J. Dermatol. Surg. Oncol. 15, 204-209.
15. GRUBB, B.R., PICKLES, R.J., YE, H., YANKASKAS, J.R., VICK, R.N., ENGELHARDT, J.F., WILSON, J.M., JOHNSON, L.G., and BOUCHER, R.C. (1994). Inefficient gene transfer by adenovirus vector to cystic fibrosis airway epithelia of mice and humans. Nature (London) 371, 802-806.
16. HAY, J.G., McELVANEY, N.G., HERENA, J., and CRYSTAL, R.G. (1995). Modification of nasal epithelial potential differences of individuals with cystic fibrosis consequent to local administration of a normal CFTR cDNA adenovirus gene transfer vector. Hum. Gene Ther. 6, 1487-1496.
17. HAYAT, M.A. (1993). Stains and Cytochemical Methods (Plenum Press, New York) pp. 202-214.
18. HIRAI, S., YASHIKI, T., and MIMA, I.L. (1981). Mechanisms for the enhancement of the nasal absorption of insulin by surfactants. Int. J. Pharm. 9, 173-184.
19. HOSOYA, K., KUBO, H., NATSUME, H., SUGIBAYASHI, K., and MORIMOTO, Y. (1994). Evaluation of enhancers to increase nasal absorption using Ussing chamber technique. Biol. Pharm. Bull. 17, 316-322.
20. JIANG, C., AKITA, G.Y., COLLEDGE, W.H., RATCLIFF, R.A., EVANS, M.J., HEHIR, K.M., ST. GEORGE, J.A., WADSWORTH, S.C., and CHENG, S.H. (1997). Increased contact time improves adenovirus-mediated CFTR gene transfer to nasal epithelium of CF mice. Hum. Gene Ther. 8, 671-680.
21. JOBE, A.H., UEDA, T., WHITSETT, J.A., TRAPNELL, B.C., and IKEGAMI, M. (1996). Surfactant enhances adenovirus-mediated gene expression in rabbit lungs. Gene Ther. 3, 775-779.
22. JOHNSON, L.G., OLSEN, J.C., SARKADI, B., MOORE, K.L., SWANSTROM, R., and BOUCHER, R.C. (1992). Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nature Genet. 2, 21-25.
23. JOHNSON, L.G., PICKLES, R.J., BOYLES, S.E., MORRIS, J.C., YE, H., ZHOU, Z., OLSEN, J.C., and BOUCHER, R.C. (1996). In vitro assessment of variables affecting the efficiency and efficacy of adenovirus-mediated gene transfer to cystic fibrosis airway epithelia. Hum. Gene Ther. 7, 51-59.
24. KATKIN, J.P., HUSSER, R.C., LANGSTON, C., and WELTY, S.E. (1997). Exogenous surfactant enhances the delivery of recombinant adenoviral vectors to the lung. Hum. Gene Ther. 8, 171-185.
25. KNOWLES, M.R., HOHNEKER, K.W., ZHOU, Z., OLSEN, J.C., NOAH, T.L., HU, P.C., LEIGH, M.W., ENGELHARDT, J.F., EDWARDS, L.J., JONES, K.R., GROSSMAN, M., WILSON, J.M., JOHNSON, L.G., and BOUCHER, R.C. (1995). A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis. N. Engl. J. Med. 333, 823-831. [See comments]
26. LIANG, E., and HUGHES, J. (1998). Characterization of a pH-sensitive surfactant, dodecyl-2-(1′-imidazolyl) propionate (DIP), and preliminary studies in liposome mediated gene transfer. Biochim. Biophys. Acta 1369, 39-50.
27. LISBY, D.A., BALLARD, P.L., FOX, W.W., WOLFSON, M.R., SHAFFER, T.H., and GONZALES, L.W. (1997). Enhanced distribution of adenovirus-mediated gene transfer to lung parenchyma by perfluorocarbon liquid. Hum. Gene Ther. 8, 919-928.
28. MERY, S., GROSS, E.A., JOYNER, D.R., GODO, M., and MORGAN, K.T. (1994). Nasal diagrams: A tool for recording the distribution of nasal lesions in rats and mice. Toxicol. Pathol. 22, 353-372.
29. MORGAN, K.T., JIANG, X.Z., PATTERSON, D.L., and GROSS, E.A. (1984). The nasal mucociliary apparatus. Correlation of structure and function in the rat. Am. Rev. Respir. Dis. 130, 275-281.
30. NIEMAN, G.F., and BRENDENBERG, C.E. (1985). High surface tension pulmonary edema induced by detergent aerosol. J. Appl. Physiol. 58, 129-136.
31. OLSEN, J.C., JOHNSON, L.G., STUTTS, M.J., SARKADI, B., YANKASKAS, J.R., SWANSTROM, R., and BOUCHER, R.C. (1992). Correction of the apical membrane chloride permeability defect in polarized cystic fibrosis airway epithelia following retroviral-mediated gene transfer. Hum. Gene Ther. 3, 253-266.
32. PARSONS, D.W., HARKEMA, J., GRUBB, B., and BOUCHER, R. (1996). Inflammation and infection is extended in the nasal airways of mice following epithelial damage. Pediatr. Pulmonol. Suppl. 13, 156.
33. PICKLES, J.R., BARKER, P.M., HONG, Y., and BOUCHER, R.C. (1996). Efficient adenovirus-mediated gene transfer to basal but not columnar cells of cartilaginous airway epithelia. Hum. Gene Ther. 7, 921-931.
34. PIEDIMONTE, G., PICKLES, R.J., LEHMANN, J.R., McCARTY, D., COSTA, D., and BOUCHER, R.C. (1997). Replication-deficient adenoviral vector for gene transfer potentiates airway neurogenic inflammation. Am. J. Respir. Cell Mol. Biol. 16, 250-258.
35. RAO, J., and DAS, P.K. (1994). Pulmonary oedema due to inhalation of detergent aerosol. Malays. J. Pathol. 16, 165-166.
36. REVEL, J.P., and KARNOVSKY, M.J. (1967). Hexagonal array of subunits in intercellular junctions of the mouse heart and liver. J. Cell Biol. 33, C7-C12.
37. RICH, D.P., ANDERSON, M.P., GREGORY, R.J., CHENG, S.H., PAUL, S., JEFFERSON, D.M., McCANN, J.D., KLINGER, K.W., SMITH, A.E., and WELSH, M.J. (1990). Expression of cystic fibrosis transmembrane conductance regulator corrects defective chloride channel regulation in cystic fibrosis airway epithelial cells. Nature (London) 347, 358-363. [See comments]
38. SALZMAN, R., MANSON, J.E., GRIFFING, G.T., KIMMERLE, R., RUDERMAN, N., McCALL, A., STOLTZ, E.I., MULLIN, C., SMALL, D., ARMSTRONG, J., and MELBY, J.C. (1985). Intranasal aerosolized insulin. Mixed-meal studies and long-term use in type I diabetes. N. Engl. J. Med. 312, 1078-1084.
39. WALKER, S., SOFIA, M.J., KAKARLA, R., KOGAN, N.A., WIERICHS, L., LONGLEY, C.B., BRUKER, K., AXELROD, H.R., MIDHA, S., BABU, S., and KAHNE, D. (1996). Cationic facial amphiphiles: A promising class of transfection agents. Proc. Natl. Acad. Sci. U.S.A. 93, 1585-1590.
40. WHEATLEY, M.A., DENT, J., WHEELDON, E.B., and SMITH, P.L. (1988). Nasal drug delivery: An in vitro characterisation of transepithelial electrical properties and fluxes in the presence or absence of enhancers. J. Controlled Release 8, 167-177.
41. WILSON, J.M. (1997). Vectors - shuttle vehicles for gene therapy. Clin. Exp. Immunol. 107(Suppl. 1):31-32.
42. WRIGHT, J.T., HALL, K.I., and GRUBB, B.R. (1996). Enamel mineral composition of normal and cystic fibrosis transgenic mice. Adv. Dent. Res. 10, 270-274.
43. YANG, Y., SU, Q., and WILSON, J.M. (1996). Role of viral antigens in destructive cellular immune responses to adenovirus vector-transduced cells in mouse lungs. J. Virol. 70, 7209-7212.
44. ZABNER, J., COUTURE, L.A., SMITH, A.E., and WELSH, M.J. (1994). Correction of cAMP-stimulated fluid secretion in cystic fibrosis airway epithelia: Efficiency of adenovirus-mediated gene transfer in vitro. Hum. Gene Ther. 5, 585-593.
45. ZABNER, J., RAMSEY, B.W., MEEKER, D.P., AITKEN, M.L., BALFOUR, R.P., GIBSON, R.L., LAUNSPACH, J., MOSCICKI, R.A., RICHARDS, S.M., STANDAERT, T.A., WILLIAMS-WARREN, J., WADSWORTH, S.C., SMITH, A.E., and WELSH, M.J. (1996a). Repeat administration of an adenovirus vector encoding cystic fibrosis transmembrane conductance regulator to the nasal epithelium of patients with cystic fibrosis. J. Clin. Invest. 97, 1504-1511.
46. ZABNER, J., ZEIHER, B.G., FRIEDMAN, E., and WELSH, M.J. (1996b). Adenovirus-mediated gene transfer to ciliated airway epithelia requires prolonged incubation time. J. Virol. 70, 6994-7003.