Lentiviral airway gene transfer in lungs of mice and sheep: Successes and challenges

Journal of Gene Medicine

Volumn 12, Issue 8, 2010, Pages 647-658

  Liu, Chuanhe ^a,b   Wong, Edward ^a,c   Miller, Darren ^a,b   Smith, Greg ^a   Anson, Don ^c,d   Parsons, David ^a,b,d  

^a WOMEN S AND CHILDREN S HOSPITAL   (Australia)

^b CHILD HEALTH RESEARCH INSTITUTE   (Australia)

^c SA Pathology ^*  (Australia)

^d UNIVERSITY OF ADELAIDE   (Australia)

Author keywords

Lentiviral vector; Lung gene transfer; Lysophosphatidylcholine; Mouse; Sheep

Indexed keywords

BETA GALACTOSIDASE; LENTIVIRUS VECTOR; LYSOPHOSPHATIDYLCHOLINE;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BASAL CELL; BRONCHOSCOPY; BRONCHUS; CELL DIFFERENTIATION; CELL TYPE; FEMALE; GENE EXPRESSION; IN VIVO STUDY; LUNG; MOUSE; NONHUMAN; PRIORITY JOURNAL; SHEEP; TRACHEA; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; FEMALE; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; LENTIVIRUS; LUNG; LYSOPHOSPHATIDYLCHOLINES; MICE; MICE, INBRED C57BL; RESPIRATORY SYSTEM; SHEEP; TRANSDUCTION, GENETIC;

ANIMALIA; MUS; OVIS ARIES; VESICULAR STOMATITIS VIRUS;

EID: 77956636830     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.1481     Document Type: Article

References (31)

1. Boucher RC. An overview of the pathogenesis of cystic fibrosis lung disease. Adv Drug Deliv Rev 2002; 54: 1359-1371.
2. Griesenbach U, Alton E. Gene transfer to the lung: Lessons learned from more than 2 decades of CF gene therapy. Adv Drug Deliv Rev 2009; 61: 128-139.
3. Anson DS. The use of retroviral vectors for gene therapy-what are the risks? A review of retroviral pathogenesis and its relevance to retroviral vector-mediated gene delivery. Genet Vaccines Ther 2004; 2: 9-21.
4. Copreni E, Penzo M, Carrabino S, et al. Lentivirus-mediated gene transfer to the respiratory epithelium: a promising approach to gene therapy of cystic fibrosis. Gene Ther 2004; 11(Suppl 1): S67-S75.
5. Stocker AG, Kremer KL, Koldej R, et al. Single-dose lentiviral gene transfer for lifetime airway gene expression. J Gene Med 2009; 11: 861-867.
6. Brunetti-Pierri N, Ng P. Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors. Gene Ther 2008; 15: 553-560.
7. McLachlan G, Baker A, Tennant P, et al. Optimizing aerosol gene delivery and expression in the ovine lung. Mol Ther 2007; 15: 348-354.
8. Beck SE, Laube BL, Barberena CI, et al. Deposition and expression of aerosolized rAAV vectors in the lungs of rhesus macaques. Mol Ther 2002; 6: 546-554.
9. Yu ZY, McKay K, Van Asperen P, et al. Lentivirus vector-mediated gene transfer to the developing bronchiolar airway epithelium in the fetal lamb. J Gene Med 2007; 9: 429-439.
10. Tarantal AF, Lee CC, Jimenez DF, et al. Fetal gene transfer using lentiviral vectors: in vivo detection of gene expression by microPET and optical imaging in fetal and infant monkeys. Hum Gene Ther 2006; 17: 1254-1261.
11. Pack RJ, Al-Ugaily LH, Morris G. The cells of the tracheobronchial epithelium of the mouse: a quantitative light and electron microscope study. J Anat 1981; 132: 71-84.
12. Grubb BR, Paradiso AM, Boucher RC. Anomalies in ion transport in CF mouse tracheal epithelium. Am J Physiol 1994; 267: C293-C300. (Pubitemid 2128165)
13. Grubb BR, Rogers TD, Boucher RC, et al. Ion transport across CF and normal murine olfactory and ciliated epithelium. Am J Physiol Cell Physiol 2009; 296: C1301-C1309.
14. Ostrowski LE, Yin W, Diggs PS, et al. Expression of CFTR from a ciliated cell-specific promoter is ineffective at correcting nasal potential difference in CFmice. Gene Ther 2007; 14: 1492-1501.
15. Choi HK, Finkbeiner WE, Widdicombe JH. A comparative study of mammalian tracheal mucous glands. J Anat 2000; 197: 361-372.
16. Anson DS, Limberis M. An improved beta-galactosidase reporter gene. J Biotechnol 2004; 108: 17-30.
17. Anson DS, Fuller M. Rational development of a HIV-1 gene therapy vector. J Gene Med 2003; 5: 829-838.
18. Koldej R, Cmielewski P, Stocker A, et al. Optimisation of a multipartite human immunodeficiency virus based vector system; control of virus infectivity and large-scale production. J Gene Med 2005; 7: 1390-1399. (Pubitemid 41672397)
19. Kremer KL, Dunning KR, Parsons DW, et al. Gene delivery to airway epithelial cells in vivo: a direct comparison of apical and basolateral transduction strategies using pseudotyped lentivirus vectors. J Gene Med 2007; 9: 362-368.
20. Dupuit F, Zahm JM, Pierrot D, et al. Regenerating cells in human airway surface epithelium represent preferential targets for recombinant adenovirus. Hum Gene Ther 1995; 6: 1185-1193.
21. Hiatt PW, Brunetti-Pierri N, McConnell R, et al. Bronchoscope-guided, targeted lobar aersolization of HDadV into the airways of nonhuman primate results in high uniform pulmonary transduction throughout the entire lung with minimal toxicity. Mol Ther 2006; 13: S264.
22. Martin GP, Marriott C, Kellaway IW. Direct effect of bile salts and phospholipids on the physical properties of mucus. Gut 1978; 19: 103-107.
23. Castellani S, Conese M. Lentiviral vectors and cystic fibrosis gene therapy. Viruses 2010; 2: 395-412.
24. Bomsel M. Transcytosis of infectious human immunodeficiency virus across a tight human epithelial cell line barrier. Nat Med 1997; 3: 42-47. (Pubitemid 27019139)
25. Di Pasquale G, Chiorini JA. AAV transcytosis through barrier epithelia and endothelium. Mol Ther 2006; 13: 506-516.
26. Kobinger GP, Limberis MP, Somanathan S, et al. Human immunodeficiency viral vector pseudotyped with the spike envelope of severe acute respiratory syndrome coronavirus transduces human airway epithelial cells and dendritic cells. Hum Gene Ther 2007; 18: 413-422.
27. Griesenbach U, Meng C, Farley R, et al. The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways. Biomaterials 31: 2665-2672.
28. Shin S, Shea LD. Lentivirus immobilization to nanoparticles for enhanced and localized delivery from hydrogels. Mol Ther 18: 700-706.
29. Ansorge S, Lanthier S, Transfiguracion J, et al. Development of a scalable process for high-yield lentiviral vector production by transient transfection of HEK293 suspension cultures. J Gene Med 2009; 11: 868-876.
30. Rogers CS DA Stoltz DK. Meyerholz, et al. Disruption of the CFTR gene produces a model of cystic fibrosis in newborn pigs. Science 2008; 321: 1837-1841.
31. Sun X, Yan Z, Yi Y, et al. Adeno-associated virus-targeted disruption of the CFTR gene in cloned ferrets. J Clin Invest 2008; 118: 1578-1583.