Efficient gene transfer to airway epithelium using recombinant Sendai virus

Yoshikazu Yonemitsu; Christopher Kitson; Stefano Ferrari; Raymond Farley; Uta Griesenbach; Diane Judd; Rachel Steel; Philippe Scheid; Jie Zhu; Peter K. Jeffery; Atsushi Kato; Mohammad K. Hasan; Yoshiyuki Nagai; Ichiro Masaki; Masayuki Fukumura; Mamoru Hasegawa; Duncan M. Geddes; Eric W.F.W. Alton

doi:10.1038/79463

Efficient gene transfer to airway epithelium using recombinant Sendai virus

Yoshikazu Yonemitsu, Christopher Kitson, Stefano Ferrari, Raymond Farley, Uta Griesenbach, Diane Judd, Rachel Steel, Philippe Scheid, Jie Zhu, Peter K. Jeffery, Atsushi Kato, Mohammad K. Hasan, Yoshiyuki Nagai, Ichiro Masaki, Masayuki Fukumura, Mamoru Hasegawa, Duncan M. Geddes, Eric W.F.W. Alton

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209 Citations (Scopus)

Abstract

Clinical studies of gene therapy for cystic fibrosis (CF) suggest that the key problem is the efficiency of gene transfer to the airway epithelium. The availability of relevant vector receptors, the transient contact time between vector and epithelium, and the barrier function of airway mucus contribute significantly to this problem. We have recently developed recombinant Sendai virus (SeV) as a new gene transfer agent. Here we show that SeV produces efficient transfection throughout the respiratory tract of both mice and ferrets in vivo, as well as in freshly obtained human nasal epithelial cells in vitro. Gene transfer efficiency was several log orders greater than with cationic liposomes or adenovirus. Even very brief contact time was sufficient to produce this effect, and levels of expression were not significantly reduced by airway mucus. Our investigations suggest that SeV may provide a useful new vector for airway gene transfer.

Original language	English
Pages (from-to)	970-973
Number of pages	4
Journal	Nature Biotechnology
Volume	18
Issue number	9
DOIs	https://doi.org/10.1038/79463
Publication status	Published - 2000
Externally published	Yes

All Science Journal Classification (ASJC) codes

Biotechnology
Bioengineering
Applied Microbiology and Biotechnology
Molecular Medicine
Biomedical Engineering

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10.1038/79463

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Yonemitsu, Y., Kitson, C., Ferrari, S., Farley, R., Griesenbach, U., Judd, D., Steel, R., Scheid, P., Zhu, J., Jeffery, P. K., Kato, A., Hasan, M. K., Nagai, Y., Masaki, I., Fukumura, M., Hasegawa, M., Geddes, D. M., & Alton, E. W. F. W. (2000). Efficient gene transfer to airway epithelium using recombinant Sendai virus. Nature Biotechnology, 18(9), 970-973. https://doi.org/10.1038/79463

Yonemitsu, Y, Kitson, C, Ferrari, S, Farley, R, Griesenbach, U, Judd, D, Steel, R, Scheid, P, Zhu, J, Jeffery, PK, Kato, A, Hasan, MK, Nagai, Y, Masaki, I, Fukumura, M, Hasegawa, M, Geddes, DM & Alton, EWFW 2000, 'Efficient gene transfer to airway epithelium using recombinant Sendai virus', Nature Biotechnology, vol. 18, no. 9, pp. 970-973. https://doi.org/10.1038/79463

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title = "Efficient gene transfer to airway epithelium using recombinant Sendai virus",

abstract = "Clinical studies of gene therapy for cystic fibrosis (CF) suggest that the key problem is the efficiency of gene transfer to the airway epithelium. The availability of relevant vector receptors, the transient contact time between vector and epithelium, and the barrier function of airway mucus contribute significantly to this problem. We have recently developed recombinant Sendai virus (SeV) as a new gene transfer agent. Here we show that SeV produces efficient transfection throughout the respiratory tract of both mice and ferrets in vivo, as well as in freshly obtained human nasal epithelial cells in vitro. Gene transfer efficiency was several log orders greater than with cationic liposomes or adenovirus. Even very brief contact time was sufficient to produce this effect, and levels of expression were not significantly reduced by airway mucus. Our investigations suggest that SeV may provide a useful new vector for airway gene transfer.",

author = "Yoshikazu Yonemitsu and Christopher Kitson and Stefano Ferrari and Raymond Farley and Uta Griesenbach and Diane Judd and Rachel Steel and Philippe Scheid and Jie Zhu and Jeffery, {Peter K.} and Atsushi Kato and Hasan, {Mohammad K.} and Yoshiyuki Nagai and Ichiro Masaki and Masayuki Fukumura and Mamoru Hasegawa and Geddes, {Duncan M.} and Alton, {Eric W.F.W.}",

note = "Funding Information: We thank Graciela Sala-Newby and Andrew Baker (Bristol Heart Institute, University of Bristol, UK) for providing the Adeno-luc, Seng Cheng (Genzyme Corp., Framingham, MA) for providing GL-67/DOPE/DMPE-PEG5000, and George Lai for help with propagating SeV. This study was supported by the Cystic Fibrosis Research Trust, the Uehara Memorial Bio-Medical Research Foundation (Y.Y.), Daiwa Anglo-Japanese Foundation (Y.Y.), a Wellcome Trust Travelling Research Fellowship (Y.Y.). and by a Wellcome Trust Senior Clinical Fellowship (E.A.).",

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AU - Kitson, Christopher

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AU - Judd, Diane

AU - Steel, Rachel

AU - Scheid, Philippe

AU - Zhu, Jie

AU - Jeffery, Peter K.

AU - Kato, Atsushi

AU - Hasan, Mohammad K.

AU - Nagai, Yoshiyuki

AU - Masaki, Ichiro

AU - Fukumura, Masayuki

AU - Hasegawa, Mamoru

AU - Geddes, Duncan M.

AU - Alton, Eric W.F.W.

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N2 - Clinical studies of gene therapy for cystic fibrosis (CF) suggest that the key problem is the efficiency of gene transfer to the airway epithelium. The availability of relevant vector receptors, the transient contact time between vector and epithelium, and the barrier function of airway mucus contribute significantly to this problem. We have recently developed recombinant Sendai virus (SeV) as a new gene transfer agent. Here we show that SeV produces efficient transfection throughout the respiratory tract of both mice and ferrets in vivo, as well as in freshly obtained human nasal epithelial cells in vitro. Gene transfer efficiency was several log orders greater than with cationic liposomes or adenovirus. Even very brief contact time was sufficient to produce this effect, and levels of expression were not significantly reduced by airway mucus. Our investigations suggest that SeV may provide a useful new vector for airway gene transfer.

AB - Clinical studies of gene therapy for cystic fibrosis (CF) suggest that the key problem is the efficiency of gene transfer to the airway epithelium. The availability of relevant vector receptors, the transient contact time between vector and epithelium, and the barrier function of airway mucus contribute significantly to this problem. We have recently developed recombinant Sendai virus (SeV) as a new gene transfer agent. Here we show that SeV produces efficient transfection throughout the respiratory tract of both mice and ferrets in vivo, as well as in freshly obtained human nasal epithelial cells in vitro. Gene transfer efficiency was several log orders greater than with cationic liposomes or adenovirus. Even very brief contact time was sufficient to produce this effect, and levels of expression were not significantly reduced by airway mucus. Our investigations suggest that SeV may provide a useful new vector for airway gene transfer.

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Efficient gene transfer to airway epithelium using recombinant Sendai virus

Abstract

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