Safety of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator cDNA to the lungs of nonhuman primates

Human Gene Therapy

Volumn 7, Issue 3, 1996, Pages 301-318

  Wilmott, Robert W ^a,c   Amin, Raouf S ^a   Perez, Carlos R ^a   Wert, Susan E ^a   Keller, Gary ^a   Boivin, Gregory P ^a   Hirsch, Raphael ^a   De Inocencio, Jaime ^a   Lu, Patrick ^b   Reising, Shirley F ^a   Yei, Soonpin ^b   Whitsett, Jeffrey A ^a   Trapnell, Bruce C ^b  

^a UNIVERSITY OF CINCINNATI COLLEGE OF MEDICINE   (United States)

^b NOVARTIS PHARMA AG   (Switzerland)

^c CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AMINOTRANSFERASE; BILIRUBIN; CD8 ANTIGEN; COMPLEMENTARY DNA; ELECTROLYTE; HEMOGLOBIN; INTERLEUKIN 1BETA; INTERLEUKIN 8; TRANSMEMBRANE CONDUCTANCE REGULATOR;

ADENOVIRUS; AMINOTRANSFERASE BLOOD LEVEL; ANIMAL EXPERIMENT; ARTICLE; ASPIRATION PNEUMONIA; BILIRUBIN BLOOD LEVEL; BLOOD CHEMISTRY; BLOOD PRESSURE; BODY TEMPERATURE; BODY WEIGHT; BRONCHOSCOPY; CLINICAL EXAMINATION; CONTROLLED STUDY; CYSTIC FIBROSIS; ELECTROLYTE BLOOD LEVEL; FEMALE; GENE THERAPY; GENE TRANSFER; HEART RATE; HEMATOLOGY; HISTOLOGY; LEUKOCYTE COUNT; LUNG; LUNG ALVEOLITIS; LUNG LAVAGE; MACACA; NONHUMAN; OXYGEN SATURATION; PRIMATE; PULSE OXIMETRY; SAFETY; TACHYPNEA; THORAX RADIOGRAPHY; TOXICITY; UREA NITROGEN BLOOD LEVEL;

ADENOVIRIDAE; ANIMALIA; MACACA; PRIMATES;

EID: 19144370024     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1996.7.3-301     Document Type: Article

References (53)

1. ALTON, E.W., MIDDLETON, P.G., CAPLEN, N.J., SMITH, S.N., STEEL, D.M., MUNKONGE, F.M., JEFFERY, P.K., GEDDES, D.M., HART, S.L., WILLIAMSON, R., FASOLD, K.I., MILLER, A.D., DICKINSON, P., STEVENSON, B.J., McLACHLAN, G., DORIN, J.R., and PORTEOUS, D.J. (1993). Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice. Nature Genet. 5, 135-142.
2. AMIN, R., WILMOTT, R., SCHWARZ, Y., TRAPNELL, B., and STARK, J. (1995). Replication-deficient adenovirus induces expression of interleukin-8 by airway epithelial cells in vitro. Hum. Gene Ther. 6, 145-153.
3. BAYLE, J.Y. JOHNSON, L.G., STGEORGE, J.A., BOUCHER, R.C., and OLSEN, J.C. (1993). High-efficiency gene transfer to primary monkey airway epithelial cells with retrovirus vectors using the gibbon ape leukemia virus receptor. Hum. Gene Ther. 4, 161-170.
4. BEAR, C.E., LI, C.H., KARTNER, N., BRIDGES, R.J., JENSEN, T.J., RAMJEESINGH, M., and RIORDAN, J.R. (1992). Purification and functional reconstitution of the cystic fibrosis transmembrane conductance regulator (CFTR). Cell 68, 809-818.
5. BOAT, T.F., WELSH, M.J., and BEAUDET, A.L. (1989). Cystic fibrosis. In the metabolic basis of inherited disease. C.R. Scriver, A.L. Beaudet, W.S. Sly, and D. Valle, eds. (McGraw-Hill, New York, NY) pp. 2649-2680.
6. BOUCHER, R.C., KNOWLES, M.R., JOHNSON, L.G., OLSEN, J.C., PICKLES, R., WILSON, J.M., ENGELHARDT, J., YANG, Y., and GROSSMAN, M. (1994). Gene therapy for cystic fibrosis using E1-deleted adenovirus: a phase I trial in the nasal cavity. The University of North Carolina at Chapel Hill. Hum. Gene Ther. 5, 615-639.
7. BRODY, S.L., METZGER, M.E., and CRYSTAL, R.G. (1993). Repeat airway administration of replication deficient recombinant adenovirus vectors to non-human primates is safe and effective. Am. Rev. Respir. Dis. 147, A544.
8. BRODY, S.L., METZGER, M., DANEL, C., ROSENFELD, M.A., and CRYSTAL, R.G. (1994). Acute responses of non-human primates to airway delivery of an adenovirus vector containing the human cystic fibrosis transmembrane conductance regulator cDNA. Hum. Gene Ther. 5, 821-836.
9. CAPLEN, N.J., ALTON, E.W.F.W., MIDDLETON, P.G., DORIN, J.R., STEVENSON, B.J., GAO, X., DURHAM, S.R., JEFFERY, P.K., HUDSON, M.E., COUTELLE, C., HUANG, L., PORTEOUS, D.J., WILLIAMSON, R., and GEDDES, D.M. (1995). Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nature Med. 1, 39-46.
10. CHIRGWIN, J.M., PRZYBYLA, A.E., MACDONALD, R.J., and RUTTER, W.J. (1979). Isolation of biologically active ribonucleic acid from sources enriched in ribonuclease. Biochemistry 18, 5294-5299.
11. CHU, C.S., TRAPNELL, B.C., CURRISTIN, S.M., CUTTING, G.R., and CRYSTAL, R.G. (1992). Extensive posttranscriptional deletion of the coding sequences for part of nucleotide-binding fold 1 in respiratory epithelial mRNA transcripts of the cystic fibrosis transmembrane conductance regulator gene is not associated with the clinical manifestat ions of cystic fibrosis. J. Clin. Invest. 90, 785-790.
12. CRYSTAL, R.G. (1992). Protocol of gene therapy of the respiratory manifestations of cystic fibrosis using a replication deficient recombinant adenovirus to transfer the normal cystic fibrosis transmembrane conductance regulator cDNA to the airway epithelium. Fed. Reg. 57, 49584.
13. 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.
14. DAI, Y.L., DEAN, T.P., CHURCH, M.K., WARNER, J.O., and SHUTE, J.K. (1994). Desensitisation of neutrophil responses by systemic interleukin 8 in cystic fibrosis. Thorax 49, 867-871.
15. 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.
16. ENGELHADT, J.F., SIMON, R.H., YANG, Y.P., ZEPEDA, M., WEBERPENDLETON, S., DORANZ, B., GROSSMAN, M., and WILSON, J.M. (1993). Adenovirus-mediated transfer of the CFTR gene to lung of nonhuman primates - biological efficacy study. Hum. Gene Ther. 4, 759-769.
17. ENGELHARDT, J.F., YE, X., DORANZ, B., and WILSON, J.M. (1994). Ablation of E2A in recombinant adenoviruses improves transgene persistence and decreases inflammatory response in mouse liver. Proc. Natl. Acad. Sci. USA 91, 6196-6200.
18. FICK, R.B., JR., NAEGEL, G.P., SQUIER, S.U., WOOD, R.E., GEE, J.B., and REYNOLDS, H.Y. (1984). Proteins of the cystic fibrosis respiratory tract. Fragmented immunoglobulin G opsonic antibody causing defective opsonophagocytosis. J. Clin. Invest. 74, 236-248.
19. FLOTTE, T.R. (1993). Prospects for virus-based gene therapy for cystic fibrosis. J. Bioenerget. Biomembr. 25, 37-42.
20. GINSBERG, H.S., HORSWOOD, R.L., CHANOCK, R.M., and PRINCE, G.A. (1990). Role of early genes in pathogenesis of adenovirus pneumonia. Proc. Natl. Acad. Sci. USA 87, 6191-6195.
21. GINSBERG, H.S., MOLDAWER, L.L., SEHGAL, P.B., REDINGTON, M., KILIAN, P.L., CHANOCK, R.M., and PRINCE, G.A. (1991). A mouse model for investigating the molecular pathogenesis of adenovirus pneumonia. Proc. Natl. Acad. Sci. USA 88, 1651-1655.
22. HIRSCH, R., ECKHAUS, M., AUCHINCLOSS, H.J., SACHS, D.H., and BLUESTONE, J.A. (1988). Effects of in vivo administration of anti-T3 monoclonal antibody on T cell function in mice. I: Immunosuppression of transplantation responses. J. Immunol. 140, 3766.
23. HORNBECK, P. (1994). Immunoassays. In Current Protocols in Molecular Biology. F.M. Ausubel, R. Brent, R.E. Kingston, D.D. Moore, J.G. Seidman, J.A. Smith, and K. Struhl, eds. (Wiley-Greene, New York) pp. 11.2.8-11.2.10.
24. 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.
25. KEREM, B., ROMMENS, J.M., BUCHANAN, J.A., MARKIEWICZ, D., COX, T.K., CHAKRAVARTI, A., BUCHWALD, M., and TSUI, L.-C. (1989). Identification of the cystic fibrosis gene: genetic analysis. Science 245, 1073-1080.
26. KUNKEL, S.L., STANDIFORD, T., KASAHARA, K., and STRIETER, R.M. (1991). Interleukin-8 (IL-8): The major neutrophil chemotactic factor in the lung. Exp. Lung Res. 17, 17-23.
27. MITTEREDER, N., YEI, S., BACHURSKI, C., CUPPOLETTI, J., WHITSETT, J.A., TOLSTOSHEV, P., and TRAPNELL, B.C. (1994). Evaluation of the efficacy and safety of in vitro, adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator cDNA. Hum. Gene Ther. 5, 717-729.
28. NAGAOKA, I., TRAPNELL, B.C., and CRYSTAL, R.G. (1990). Upregulation of platelet-derived growth factor-A and -B gene expression in alveolar macrophages of individuals with idiopathic pulmonary fibrosis. J. Clin. Invest. 85, 2023-2027.
29. RENNARD, S.I., BASSET, B., LECOSSIER, D., O'DONNELL, K.M., PINKSTON, P., MARTIN, M., and CRYSTAL, R.G. (1986). Estimation of volume of epithelial lining fluid recovered by lavage using urea as marker of dilution. J. Appl. Physiol. 60, 532-538.
30. 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 defecive chloride channel regulation in cystic fibrosis airway epithelial cells [see comments]. Nature 347, 358-363.
31. RICH, D.P., COUTURE, L.A., CARDOZA, L.M., GUIGGIO, V.M., ARMENTANO, D., ESPINO, P.C., HEHIR, K., WELSH, M .J., SMITH, A.E., and GREGORY, R.J. (1993). Development and analysis of recombinant adenoviruses for gene therapy of cystic fibrosis. Hum. Gene Ther. 4, 461-576.
32. RIORDAN, J.R., ROMMENS, J.M., KEREM, B., ALON, N., ROZMAHEL, R., GRZELCZAK, Z., ZIELENSKI, J., LOK, S., PLAVSIC, N., CHOU, J.L., DRUMM, M.L., IANNUZZI, M.C., COLLINS, F.S., and TSUI, L.C. (1989). Identification of the cystic fibrosis gene: Cloning and characterization of complementary DNA. Science 245, 1066-1073.
33. ROMMENS, J.M., IANNUZZI, M.C., KEREM, B., DRUMM, M.L., MELMER, G., DEAN, M., ROZMAHEL, R., COLE, J.L., KENNEDY, D., HIDAKA, N., ZSIGA, M., BUCHWALD, M., RIORDAN, J.R., TSUI, L.C. and COLLINS, F.S. (1989). Identification of the cystic fibrosis gene: chromosome walking and jumping. Science 245, 1059-1065.
34. ROSENFELD, M.A., YOSHIMURA, K., TRAPNELL, B.C., YONEYAMA, K., ROSENTHAL, E.R., DALEMANS, W., FUKAYAMA, M., BARGON, J., STIER, L.E., STRATFORD PERRICAUDET, L., PERRICAUDET, M., GUGGINO, W.B., PAVIRANI, A., LECOCQ, J.P., and CRYSTAL, R.G. (1992). In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium. Cell 68, 143-155.
35. ROSENFELD, M.A., RONALD, G., and CRYSTAL, R.G. (1993). Gene therapy for pulmonary diseases. Pathol. Biol. 41, 677-680.
36. SIMON, R.H., ENGELHARDT, J.F., YANG, Y., ZEPEDA, M., WEBER PENDLETON, S., GROSSMAN, M., and WILSON, J.M. (1993). Adenovirus-mediated transfer of the CFTR gene to lung of nonhuman primates: Toxicity study. Hum. Gene Ther. 4, 771-780.
37. SORSCHER, E.J., and LOGAN, J.J. (1994). Gene therapy for cystic fibrosis using cationic liposome mediated gene transfer: A phase I trial of safety and efficacy in the nasal airway. Hum. Gene Ther. 5, 1259-1277.
38. TRAPNELL, B.C. (1993). Adenoviral vectors for gene transfer. Adv. Drug Delivery Rev. 12, 185-199.
39. TRAPNELL, B.C., YEI, S., WERT, S., MITTEREDER, N., and WHITSETT, J.A. (1993). Evaluation of the host response to in vivo adenovirus-mediated gene transfer. Pediatr. Pulmonol. S.9, 241.
40. WELSH, M.J. (1992). Cystic fibrosis gene therapy using an adenovirus vector: In vivo safety and efficacy in the nasal epithelium. Fed. Reg. 57, 49584.
41. WELSH, M.J., SMITH, A.E., ZABNER, J., RICH, D.P., GRAHAM, S.M., GREGORY, R.J., PRATT, B.M., and MOSCICKI, R.A. (1994). Cystic fibrosis gene therapy using an adenovirus vector: in vivo safety and efficacy in nasal epithelium. Hum. Gene Ther. 5, 209-219.
42. WHITSETT, J.A., DEY, C.R., STRIPP, B.R., WIKENHEISER, K.A., CLARK, J.C., WERT, S.E., GREGORY, R.J., SMITH, A.E., COHN, J.A., WILSON, J.M., and ENGELHARDT, J. (1992). Human cystic fibrosis transmembrane conductance regulator directed to respiratory epithelial cells of transgenic mice. Nature Genet. 2, 13-20.
43. WILMOTT, R.W., and FIEDLER, M.A. (1994). Recent advances in the treatment of cystic fibrosis. Pediatr. Clin. North Am. 41, 431-451.
44. WILMOTT, R.W., WHITSETT, J.A., TRAPNELL, B., WERT, S., BAUGHMAN, R., CUPPOLETTI, J., and TOLSTOHEV, P. (1994). Gene therapy for cystic fibrosis utilizing a replication deficient recombinant adenovirus vector to deliver the human cystic fibrosis transmembrane conductance regulator cDNA to the airways. A phase I study. Hum. Gene Ther. 5, 1019-1057.
45. WILSON, J.M., ENGELHARDT, J.F., GROSSMAN, M., SIMON, R.H., and YANG, Y. (1994). Gene therapy of cystic fibrosis lung disease using E1 deleted adenoviruses: a phase I trial. Hum. Gene Ther. 5, 501-519.
46. WOOD, R.E., BOAT, T.F., and DOERSHUK, C.F. (1976). Cystic fibrosis: State of the art. Am. Rev. Respir. Dis. 113, 833-878.
47. YANG, Y.P., ERTL, H.C.J., and WILSON, J.M. (1994). MHC class I-restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1-deleted recombinant adenoviruses. Immunity 1, 433-442.
48. YEI, S., MITTEREDER, N., WERT, S., WHITSETT, J.A., WILMOTT, R.W., and TRAPNELL, B.C. (1994). In vivo evaluation of the safety of adenovirus-mediated transfer of human cystic fibrosis transmembrane conductance regulator cDNA to the lung. Hum. Gene Ther. 5, 731-744.
49. YEI, S., MITTEREDER, N., WHITSETT, J.A., WILMOTT, R.W., and TRAPNELL, B.C. (1994). The role of the host response in adenovirus vector-mediated gene therapy for cystic fibrosis lung disease: Toxicology and pharmacokinetics. Pediatr. Pulmonol. (Suppl.) 10, 151-152.
50. YOSHIMURA, K., NAKAMURA, H., TRAPNELL, B.C., DALEMANS, W., PAVIRANI, A., LECOCQ, J.P., and CRYSTAL, R.G. (1991). The cystic fibrosis gene has a 'housekeeping'-type promoter and is expressed at low levels in cells of epithelial origin. J. Biol. Chem. 266, 9140-9144.
51. ZABNER, J., COUTURE, L.A., GREGORY, R.J., GRAHAM, S.M., SMITH, A.E., and WELSH, M.J. (1993). Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell 75, 207-216.
52. ZABNER, J., PETERSEN, D.M., PUGA, A.P., GRAHAM S.M., COUTURE, L.A., KEYES, L.D., LUKASON, M.J., ST.GEORGE, J.A., GREGORY, R.J., SMITH, A.E., and WELSH, M.J. (1994). Safety and efficacy of repetitive adenovirus-mediated transfer of CFTR cDNA to airway epithelia of primates and cotton rats. Nature Genet. 6, 75-83.
53. ZSENGELLER, Z.K., WERT, S., HULL, W.M., HU, X., YEI, S., TRAPNELL, B., and WHITSETT, J.A. (1995). Persistence of replication-deficient adenovirus-mediated gene transfer in lungs of immune deficient (NU/NU) mice. Hum. Gene Ther. 6, 457-467.