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

Human Gene Therapy

Volumn 10, Issue 18, 1999, Pages 2973-2985

  Zuckerman, Jonathan B ^a,e   Robinson, Cynthia B ^b   McCoy, Karen S ^c   Shell, Richard ^c   Sferra, Thomas J ^a   Chirmule, Narendra ^a   Magosin, Susan A ^a   Propert, Kathleen J ^a   Brown Parr, Elsbeth C ^a   Hughes, Joseph V ^a   Tazelaar, John ^a   Baker, Colleen ^a   Goldman, Mitchell J ^d   Wilson, James M ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b SmithKline Beecham   (United States)

^c OHIO STATE UNIVERSITY   (United States)

^d SANOFI   (France)

^e MAINE MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

TRANSMEMBRANE CONDUCTANCE REGULATOR; VIRUS VECTOR;

ADULT; ARTICLE; CLINICAL ARTICLE; CYSTIC FIBROSIS; FEMALE; GENE TRANSFER; HUMAN; MALE; REGULATOR GENE; RESPIRATORY EPITHELIUM;

ADENOVIRIDAE; BASE SEQUENCE; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DNA PROBES; GENE THERAPY; GENE TRANSFER TECHNIQUES; HUMANS; LUNG; NEUTRALIZATION TESTS;

ADENOVIRIDAE;

EID: 0033544951     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430349950016384     Document Type: Article

References (21)

1. ARMENTANO, D., ZABNER, J., SACKS, C., SOOKDEO, C.C., SMITH, M.P., ST. GEORGE, J.A., WADWORTH, S.C., SMITH, A.E., and GREGORY, R.J. (1997). Effect of the E4 region on the persistence of transgene expression from adenovirus vectors. J. Virol. 71, 2408-2416.
2. BERGELSON, J.M., CUNNINGHAM, J.A., DROGUETT, G., KURT-JONES, E.A., KRITHIVAS, A., HONG, J.S., HORWITZ, M.S., CROWELL, R.L., and FINBERG, R.W. (1997). Isolation of a common receptor for coxsackie B viruses and adenoviruses 2 and 5. Science 275, 1320-1323.
3. CYSTIC FIBROSIS FOUNDATION (1995). Patient Registry Annual Data Report 1995. (Cystic Fibrosis Foundation, Bethesda, MD).
4. CHENG, P.W., BOAT, T.F., CRANFILL, K., YANKASKAS, J.R., and BOUCHER, R.C. (1989). Increased sulfation of glycoconjugates by cultured nasal epithelia cells from patients with cystic fibrosis. J. Clin. Invest. 84, 68-72.
5. CHIRMULE, N., HUGHES, J.V., GAO, G.P., RAPER, S.E., and WILSON, J.M. (1998). Role of E4 in eliciting CD4 T-cell and B-cell responses to adenovirus vectors delivered to murine and nonhuman primate lungs. J. Virol. 72, 6138-6145.
6. CHIRMULE, N., PROPERT, K., MAGOSIN, S., QIAN, Y., QIAN, R., and WILSON, J.M. (1999). Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther. 6, 1574-1583.
7. CRYSTAL, R.G., McELVANEY, N.G., ROSENFELD, M.A., CHU, C., 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.
8. DEDIEU, J.F., VIGNE, E., TORRENT, C., JULLIEN, C., MAHFOUZ, I., CAILAUD, J.M., AUBAILLY, N., ORSINI, C., GUILLAUME, J.M., OPOLON, P., DELAERE, P., PERRICAUDET, M., and YEH, P. (1997). Long-term gene delivery into the livers of immunocompetent mice with E1/E4 defective adenoviruses. J. Virol. 71, 4626-4637.
9. GAO, G.P., YANG, Y.P., and WILSON, J.M. (1996). Biology of adenoviral vectors deleted of E1 and E4 for liver-directed gene therapy. J. Virol. 70, 8934-8943.
10. GOLDMAN, M.J., and WILSON, J.M. (1995). Expression of alphav-beta5 integrin is necessary for efficient adenovirus-mediated gene transfer in the human airway. J. Virol. 69, 5951-5958.
11. GOLDMAN, M., YANG, Y., and WILSON, J. (1995). Gene therapy in a xenograft model of cystic fibrosis lung corrects choloride transport more effectively than the sodium defect. Nature Genet. 9, 126-131.
12. GOLDMAN, M.J., ANDERSON, G.M., STOLZENBERG, E.D., KARI, U.P., ZAZLOFF, M., and WILSON, J.M. (1997). Human β-defensin-1 is a salt sensitive antibiotic in lung that is inactivated in cystic fibrosis. Cell 88, 553-560.
13. JOHNSON, L., BOYLES, S., WILSON, J., and BOUCHER, R. (1995). Normalization of raised sodium absorption and raised calcium-mediated chloride secretion by adenovirus-mediated expression of cystic fibrosis transmembrane conductance regulator in primary human cystic fibrosis airway epithelial cells. J. Clin. Invest. 95, 1377-1382.
14. KHAN, T.Z., WAGENER, J.S., BOST, T., MARTINEZ, J., ACCURSO, F.J., and RICHES, D.W. (1995). Early pulmonary inflammation in infants with cystic fibrosis. Am. J. Respir. Crit. Care Med. 151, 1075-1082.
15. KNOWLES, M.R., HOHNEKER, K.W., ZHOU, Z., OLSEN, J.C., NOAH, T.L., HU, P., 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.
16. 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., 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.
17. SMITH, J.J., TRAVIS, S.M., GREENBERG, E.P., and WELSH, M.J. (1996). Cystic fibrosis airway epithelial fail to kill bacteria because of abnormal airway surface fluid. Cell 85, 229-236.
18. 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.
19. 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.
20. 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. (1996). 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.
21. ZHANG, Y., DORANZ, B., YANKASKAS, J.R., and ENGELHARDT, J.F. (1995). Genotopic analysis of respiratory mucous sulfation defects in cystic fibrosis. J. Clin. Invest. 96, 2997-3004.