Lentiviral vectors and cystic fibrosis gene therapy

Viruses

Volumn 2, Issue 2, 2010, Pages 395-412

  Castellani, Stefano ^a   Conese, Massimo ^a  

^a UNIVERSITY OF FOGGIA   (Italy)

Author keywords

Airway epithelium; Cystic fibrosis transmembrane conductance regulator (CFTR); Lentivirus; Lung; Retroviridae

Indexed keywords

ADENOVIRUS VECTOR; BETA GALACTOSIDASE; CLARA CELL PROTEIN; EGTAZIC ACID; GLYCOSAMINOGLYCAN; LENTIVIRUS VECTOR; LYSOPHOSPHATIDYLCHOLINE; PARVOVIRUS VECTOR; SULFUR DIOXIDE; SURFACTANT PROTEIN C; TRANSMEMBRANE CONDUCTANCE REGULATOR; VIRUS GLYCOPROTEIN;

CLINICAL TRIAL (TOPIC); CYSTIC FIBROSIS; GENE EXPRESSION; GENETIC TRANSDUCTION; HUMAN; IMMUNE RESPONSE; IMMUNOGENICITY; INNATE IMMUNITY; LUNG DISEASE; MEMBRANE PERMEABILITY; NONHUMAN; PSEUDOMONAS AERUGINOSA; RESPIRATORY EPITHELIUM; REVIEW; SAFETY; T LYMPHOCYTE; TIGHT JUNCTION; TRANSGENE; VESICULAR STOMATITIS VIRUS; VIRAL GENE DELIVERY SYSTEM; VIRUS DNA CELL DNA INTERACTION;

ANIMALIA; LENTIVIRUS; MUS; MUSTELA; RETROVIRIDAE; SUIDAE;

EID: 77956628230     PISSN: None     EISSN: 19994915     Source Type: Journal    
DOI: 10.3390/v2020395     Document Type: Review

References (90)

1. Farrell, P.M.; Rosenstein, B.J.; White, T.B.; Accurso, F.J.; Castellani, C.; Cutting, G.R.; Durie, P.R.; Legrys, V.A.; Massie, J.; Parad, R.B.; Rock, M.J.; Campbell, P.W. Guidelines for diagnosis of cystic fibrosis in newborns through older adults: Cystic Fibrosis Foundation consensus report. J. Pediatr. 2008, 153, S4-S14.
2. 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.; Tsui, L.-C. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science 1989, 245, 1066-1073.
3. Kerem, B.; Rommens, J.M.; Buchanan, J.A.; Markiewicz, D.; Cox, T.K.; Chakravarti, A.; Buchwald, M.; Tsui, L.C. Identification of the cystic fibrosis gene: genetic analysis. Science 1989, 245, 1073-1080.
4. Drumm, M.L.; Pope, H.A.; Cliff, W.H.; Rommens, J.M.; Marvin, S.A.; Tsui, L.-C.; Collins, F.S.; Frizzel, R.A.; Wilson, J.M. Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer. Cell 1990, 62, 1227-1233.
5. Bear, C.E.; Li, C.; Kartner, N.; Bridges, R.D.; Jensen, T.J.; Ramjeesingh, M.; Riordan, J.R. Purification and functional reconstitution of the cystic fibrosis transmembrane conductance regulator (CFTR). Cell 1992, 68, 809-818.
6. Chmiel, J.F.; Berger, M.; Konstan, M.W. The role of inflammation in the pathophysiology of CF lung disease. Clin. Rev. Allergy Immunol. 2002, 23, 5-27.
7. Ratjen, F.; Doring, G. Cystic fibrosis. The Lancet 2003, 361, 681-689.
8. Boucher, R.C. New concepts of the pathogenesis of cystic fibrosis lung disease. Eur. Respir. J. 2004, 23, 146-158.
9. Rowe, S.M.; Miller, S.; Sorscher, E.J. Cystic Fibrosis. N. Engl. J. Med. 2005, 352, 1992-2001.
10. Matsui, H.; Grubb, B.R.; Tarran, R.; Randell, S.H.; Gatzy, J.T.; Davis, C.W.; Boucher, R.C. Evidence for periciliary liquid layer depletion, not abnormal ion composition, in the pathogenesis of cystic fibrosis airway disease. Cell 1998, 95, 1005-1015.
11. Mall, M.; Hipper, A.; Greger, R.; Kunzelmann, K. Wild type but not deltaF508 CFTR inhibits Na+ conductance when coexpressed in Xenopus oocytes. FEBS Lett. 1996, 381, 47-52.
12. + absorption produces cystic fibrosis-like lung disease in mice. Nat. Med. 2004, 10, 487-493.
13. Johnson, L.; Olsen, J.; Sarkadi, B.; Moore, K.; Swanstrom, R.; Boucher, R. Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nat. Genet. 1992, 2, 21-25.
14. Dorin, J.R.; Farley, R.; Webb, S.; Smith, S.N.; Farini, E.; Delaney, S.J.; Wainwright, B.J.; Alton, E.W.; Porteous, D.J. A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction. Gene Ther. 1996, 3, 797-801.
15. Farmen, S.L.; Karp, P.H.; Ng, P.; Palmer, D.J.; Koehler, D.R.; Hu, J.; Beaudet, A.L.; Zabner, J.; Welsh, M.J. Gene transfer of CFTR to airway epithelia: low levels of expression are sufficient to correct Cl- transport and overexpression can generate basolateral CFTR. Am. J. Physiol. Lung Cell Mol. Physiol. 2005, 289, L1123-1130.
16. Johnson, L.G.; Boyles, S.E.; Wilson, J.; Boucher, R.C. 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. 1995, 95, 1377-1382.
17. Goldman, M.J.; Yang, Y.; Wilson, J.M. Gene therapy in a xenograft model of cystic fibrosis lung corrects chloride transport more effectively than the sodium defect. Nat. Genet. 1995, 9, 126-131.
18. Flotte, T.R.; Laube, B.L. Gene therapy in cystic fibrosis. Chest 2001, 120, 124S-131S.
19. Driskell, R.A.; Engelhardt, J.F. Current status of gene therapy for inherited lung diseases. Annu. Rev. Physiol. 2003, 65, 585-612.
20. Lee, T.W.; Matthews, D.A.; Blair, G.E. Novel molecular approaches to cystic fibrosis gene therapy. Biochem. J. 2005, 387, 1-15.
21. Flotte, T.R. Adeno-associated virus-based gene therapy for inherited disorders. Pediatr. Res. 2005, 58, 1143-1147.
22. Flotte, T.R.; Ng, P.; Dylla, D.E.; McCray, P.B., Jr.; Wang, G.; Kolls, J.K.; Hu, J. Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis. Mol. Ther. 2007, 15, 229-241.
23. Copreni, E.; Penzo, M.; Carrabino, S.; Conese, M. Lentiviral-mediated gene transfer to the respiratory epithelium: a promising approach to gene therapy of Cystic Fibrosis. Gene Ther. 2004, 11, S67-S75.
24. Zaiss, A.K.; Son, S.; Chang, L.J. RNA 3' readthrough of oncoretrovirus and lentivirus: implications for vector safety and efficacy. J. Virol. 2002, 76, 7209-7219.
25. Naldini, L.; Blomer, U.; Gage, F.H.; Trono, D.; Verma, I.M. Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc. Natl. Acad. Sci. USA 1996, 93, 11382-11388.
26. Naldini, L.; Blomer, U.; Gallay, P.; Ory, D.; Mulligan, R.; Gage, F.H.; Verma, I.M.; Trono, D. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996, 272, 263-267.
27. Delenda, C. Lentiviral vectors: optimization of packaging, transduction and gene expression. J. Gene Med. 2004, 6 (Suppl. 1), S125-138.
28. Follenzi, A.; Ailles, L.E.; Bakovic, S.; Geuna, M.; Naldini, L. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat. Genet. 2000, 25, 217-222.
29. Zennou, V.; Petit, C.; Guetard, D.; Nerhbass, U.; Montagnier, L.; Charneau, P. HIV-1 genome nuclear import is mediated by a central DNA flap. Cell 2000, 101, 173-185.
30. Zufferey, R.; Donello, J.E.; Trono, D.; Hope, T.J. Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J. Virol. 1999, 73, 2886-2892.
31. Zufferey, R.; Dull, T.; Mandel, R.J.; Bukovsky, A.; Quiroz, D.; Naldini, L.; Trono, D. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J. Virol. 1998, 72, 9873-9880.
32. Conese, M.; Castellani, S.; Palmieri, L.; Copreni, E. Lentivirus expression vectors. In Virus Expression vectors, Research Signpost Transworld Research Network: 2007; pp 81-122.
33. Levine, B.L.; Humeau, L.M.; Boyer, J.; MacGregor, R.R.; Rebello, T.; Lu, X.; Binder, G.K.; Slepushkin, V.; Lemiale, F.; Mascola, J.R.; Bushman, F.D.; Dropulic, B.; June, C.H. Gene transfer in humans using a conditionally replicating lentiviral vector. Proc. Natl. Acad. Sci. USA 2006, 103, 17372-17377.
34. Griesenbach, U.; Ferrari, S.; Geddes, D.M.; Alton, E.W. Gene therapy progress and prospects: cystic fibrosis. Gene Ther. 2002, 9, 1344-1350.
35. Conese, M.; Copreni, E.; Piro, D.; Rejman, J. Gene and Cell Therapy for the Treatment of Cystic Fibrosis. Adv. Gene Mol. Cell Ther. 2007, 1, 99-119.
36. Ferrari, S.; Griesenbach, U.; Geddes, D.M.; Alton, E. Immunological hurdles to lung gene therapy. Clin. Exp. Immunol. 2003, 132, 1-8.
37. Duan, D.; Yue, Y.; Yan, Z.; McCray, P.B.; Engelhardt, J.F. Polarity influences the efficiency of recombinant adenoassociate virus infection in differentiated airway epithelia. Hum. Gene Ther. 1998, 9, 2761-2776.
38. Wang, G.; Davidson, B.L.; Melchert, P.; Sleupushkin, V.A.; van Hes, H.H.G.; Bodner, M.; Jolly, D.J.; McCray, P.B., Jr. Influence of cell polarity on retrovirus-mediated gene transfer to differentiated human airway epithelia. J. Virol. 1998, 72, 9818-9826.
39. Walters, R.W.; Grunst, T.; Bergelson, J.M.; Finberg, R.W.; Welsh, M.J.; Zabner, J. Basolateral localization of fiber receptor limits adenovirus infection from the apical surface of airway epithelia. J. Biol. Chem. 1999, 274, 10219-10226.
40. Bragonzi, A.; Worlitzsch, D.; Pier, G.B.; Timpert, P.; Ulrich, M.; Hentzer, M.; Andersen, J.B.; Givskov, M.; Conese, M.; Doring, G. Nonmucoid Pseudomonas aeruginosa expresses alginate in the lungs of patients with cystic fibrosis and in a mouse model. J. Infect. Dis. 2005, 192, 410-419.
41. van Heeckeren, A.; Ferkol, T.; Tosi, M. Effects of bronchopulmonary inflammation induced by pseudomonas aeruginosa on adenovirus-mediated gene transfer to airway epithelial cells in mice. Gene Ther. 1998, 5, 345-351.
42. Tosi, M.F.; van Heeckeren, A.; Ferkol, T.W.; Askew, D.; Harding, C.V.; Kaplan, J.M. Effect of Pseudomonas-induced chronic lung inflammation on specific cytotoxic T-cell responses to adenoviral vectors in mice. Gene Ther. 2004, 11, 1427-1433.
43. Rejman, J.; De Fino, I.; Paroni, M.; Bragonzi, A.; Demeester, J.; De Smedt, S.; Conese, M. The impact of chronic pulmonary infection by Pseudomonas aeruginosa on transfection mediated by viral and non-viral vectors. Hum. Gene Ther. 2010, doi:10.1089/hum.2009.085.
44. Goldman, M.J.; Lee, P.S.; Yang, J.S.; Wilson, J.M. Lentiviral vectors for Gene Therapy of cystic fibrosis. Hum. Gene Ther. 1997, 8, 2261-2268.
45. Castillon, N.; Avril-Delplanque, A.; Coraux, C.; Delenda, C.; Peault, B.; Danos, O.; Puchelle, E. Regeneration of a well-differentiated human airway surface epithelium by spheroid and lentivirus vector-transduced airway cells. J. Gene Med. 2004, 6, 846-856.
46. Lim, F.Y.; Kobinger, G.P.; Weiner, D.J.; Radu, A.; Wilson, J.M.; Crombleholme, T.M. Human fetal trachea-scid mouse xenografts: Efficacy of vesicular stomatitis virus-G pseudotyped lentiviral-mediated gene transfer. J. Pediatr. Surg. 2003, 38, 834-839.
47. Wang, G.; Slepushkin, V.; Zabner, J.; Keshavjee, S.; Johnston, J.C.; Sauter, S.L.; Jolly, D.J.; Dubensky, T.W., Jr.; Davidson, B.L.; McCray, P.B., Jr. Feline immunodeficiency virus vectors persistently transduce nondividing airway epithelia and correct the cystic fibrosis defect. J. Clin. Invest. 1999, 104, R55-62.
48. Johnson, L.G.; Olsen, J.C.; Naldini, L.; Boucher, R. Pseudotyped human lentiviral vector-mediated gene transfer to airway epithelia in vivo. Gene Ther. 2000, 7, 568-574.
49. Limberis, M.; Anson, D.S.; Fuller, M.; Parsons, D.W. Recovery of airway cystic fibrosis transmembrane conductance regulator function in mice with cystic fibrosis after single-dose lentivirus-mediated gene transfer. Hum. Gene Ther. 2002, 13, 1961-1970.
50. Rejman, J.; Di Gioia, S.; Bragonzi, A.; Conese, M. Pseudomonas aeruginosa infection destroys the barrier function of lung epithelium and enhances polyplex-mediated transfection. Hum. Gene Ther. 2007, 18, 642-652.
51. Copreni, E.; Castellani, S.; Palmieri, L.; Penzo, M.; Conese, M. Involvement of glycosaminoglycans in vesicular stomatitis virus G glycoprotein pseudotyped lentiviral vector-mediated gene transfer into airway epithelial cells. J. Gene Med. 2008, 10, 1294-1302.
52. Kremer, K.L.; Dunning, K.R.; Parsons, D.W.; Anson, D.S. 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.
53. Stocker, A.G.; Kremer, K.L.; Koldej, R.; Miller, D.S.; Anson, D.S.; Parsons, D.W. Single-dose lentiviral gene transfer for lifetime airway gene expression. J. Gene Med. 2009, 11, 861-867.
54. Borthwick, D.W.; Shahbazian, M.; Krantz, Q.T.; Dorin, J.R.; Randell, S.H. Evidence for stem-cell niches in the tracheal epithelium. Am. J. Respir. Cell Mol. Biol. 2001, 24, 662-670.
55. Rawlins, E.L.; Hogan, B.L. Ciliated epithelial cell lifespan in the mouse trachea and lung. Am. J. Physiol. Lung Cell Mol. Physiol. 2008, 295, L231-234.
56. Kobinger, G.P.; Weiner, D.J.; Yu, Q.C.; Wilson, J.M. Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo. Nat. Biotechnol. 2001, 19, 225-230.
57. Medina, M.F.; Kobinger, G.P.; Rux, J.; Gasmi, M.; Looney, D.J.; Bates, P.; Wilson, J.M. Lentiviral vectors pseudotyped with minimal filovirus envelopes increased gene transfer in murine lung. Mol. Ther. 2003, 8, 777-789.
58. Sinn, P.L.; Burnight, E.R.; Hickey, M.A.; Blissard, G.W.; McCray, P.B., Jr. Persistent gene expression in mouse nasal epithelia following feline immunodeficiency virus-based vector gene transfer. J. Virol. 2005, 79, 12818-12827.
59. McKay, T.; Patel, M.; Pickles, R.J.; Johnson, L.G.; Olsen, J.C. Influenza M2 envelope protein augments avian influenza hemagglutinin pseudotyping of lentiviral vectors. Gene Ther. 2006, 13, 715-724.
60. Kang, Y.; Stein, C.S.; Heth, J.A.; Sinn, P.L.; Penisten, A.K.; Staber, P.D.; Ratcliff, K.L.; Shen, S.; Barker, C.K.; Martins, I.; Sharkey, C.M.; Sanders, D.A.; McCray, P.B., Jr.; Davidson, B.L. In vivo gene transfer using a nonprimate lentiviral vector pseudotyped with Ross River virus glycoproteins. J. Virol. 2002, 76, 9378-9388.
61. Sinn, P.L.; Penisten, A.K.; Burnight, E.R.; Hickey, M.A.; Williams, G.; McCoy, D.M.; Mallampalli, R.K.; McCray, P.B. Gene transfer to respiratory epithelia with lentivirus pseudotyped with Jaagsiekte sheep retrovirus envelope glycoprotein. Hum. Gene Ther. 2005, 16, 479-488.
62. Huang, X.; Yang, Y. Innate immune recognition of viruses and viral vectors. Hum. Gene Ther. 2009, 20, 293-301.
63. Yang, Y.; Su, Q.; Wilson, J.M. Role of viral antigens in destructive cellular immune responses to adenovirus vector-trasduced cells in mouse lungs. J. Virol. 1996, 70, 7209-7212.
64. VandenDriessche, T.; Thorrez, L.; Naldini, L.; Follenzi, A.; Moons, L.; Berneman, Z.; Collen, D.; Chuah, M.K.L. Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo. Blood 2002, 100, 813-822.
65. Follenzi, A.; Battaglia, M.; Lombardo, A.; Annoni, A.; Roncarolo, M.G.; Naldini, L. Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice. Blood 2004, 103, 3700-3709.
66. Auricchio, A.; Kobinger, G.; Anand, V.; Hildinger, M.; O'Connor, E.; Maguire, A.M.; Wilson, J.M.; Bennett, J. Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model. Hum. Mol. Genet. 2001, 10, 3075-3081.
67. Kafri, T.; Blomer, U.; Peterson, D.A.; Gage, F.H.; Verma, I.M. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat. Genet. 1997, 17, 314-317.
68. Baekelandt, V.; Eggermont, K.; Michiels, M.; Nuttin, B.; Debyser, Z. Optimized lentiviral vector production and purification procedure prevents immune response after transduction of mouse brain. Gene Ther. 2003, 10, 1933-1940.
69. Sinn, P.L.; Arias, A.C.; Brogden, K.A.; McCray, P.B., Jr. Lentivirus vector can be readministered to nasal epithelia without blocking immune responses. J. Virol. 2008, 82, 10684-10692.
70. Limberis, M.P.; Bell, C.L.; Heath, J.; Wilson, J.M. Activation of Transgene-specific T Cells Following Lentivirus-mediated Gene Delivery to Mouse Lung. Mol. Ther. 2009, 15, 1694-1700.
71. Figueredo, J.; Limberis, M.P.; Wilson, J.M. Prediction of cellular immune responses against CFTR in patients with cystic fibrosis after gene therapy. Am. J. Respir. Cell Mol. Biol. 2007, 36, 529-533.
72. Limberis, M.P.; Figueredo, J.; Calcedo, R.; Wilson, J.M. Activation of CFTR-specific T Cells in cystic fibrosis mice following gene transfer. Mol. Ther. 2007, 15, 1694-1700.
73. Challita, P.M.; Kohn, D.B. Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo. Proc. Natl. Acad. Sci. USA 1994, 91, 2567-2571.
74. Bowtell, D.D.; Johnson, G.R.; Kelso, A.; Cory, S. Expression of genes transferred to haemopoietic stem cells by recombinant retroviruses. Mol. Biol. Med. 1987, 4, 229-250.
75. Hacein-Bey-Abina, S.; Von Kalle, C.; Schmidt, M.; McCormack, M.P.; Wulffraat, N.; Leboulch, P.; Lim, A.; Osborne, C.S.; Pawliuk, R.; Morillon, E.; Sorensen, R.; Forster, A.; Fraser, P.; Cohen, J.I.; de Saint Basile, G.; Alexander, I.; Wintergerst, U.; Frebourg, T.; Aurias, A.; Stoppa-Lyonnet, D.; Romana, S.; Radford-Weiss, I.; Gross, F.; Valensi, F.; Delabesse, E.; Macintyre, E.; Sigaux, F.; Soulier, J.; Leiva, L.E.; Wissler, M.; Prinz, C.; Rabbitts, T.H.; Le Deist, F.; Fischer, A.; Cavazzana-Calvo, M. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003, 302, 415-419.
76. Hendrickson, B.; Senadheera, D.; Mishra, S.; Bui, K.C.; Wang, X.; Chan, B.; Petersen, D.; Pepper, K.; Lutzko, C. Development of lentiviral vectors with regulated respiratory epithelial expression in vivo. Am. J. Respir. Cell Mol. Biol. 2007, 37, 414-423.
77. Diamond, G.; Legarda, D.; Ryan, L.K. The innate immune response of the respiratory epithelium. Immunol. Rev. 2000, 173, 27-38.
78. Bals, R.; Hiemstra, P.S. Innate immunity in the lung: how epithelial cells fight against respiratory pathogens. Eur. Respir. J. 2004, 23, 327-333.
79. Kato, A.; Schleimer, R.P. Beyond inflammation: airway epithelial cells are at the interface of innate and adaptive immunity. Curr. Opin. Immunol. 2007, 19, 711-720.
80. Message, S.D.; Johnston, S.L. Host defense function of the airway epithelium in health and disease: clinical background. J. Leukoc. Biol. 2004, 75, 5-17.
81. Wang, J.P.; Kurt-Jones, E.A.; Finberg, R.W. Innate immunity to respiratory viruses. Cell Microbiol. 2007, 9, 1641-1646.
82. Copreni, E.; Nicolis, E.; Tamanini, A.; Bezzerri, V.; Castellani, S.; Palmieri, L.; Giri, M.G.; Vella, A.; Colombatti, M.; Rizzotti, P.; Conese, M.; Cabrini, G. Late generation lentiviral vectors: Evaluation of inflammatory potential in human airway epithelial cells. Virus Res. 2009, 144, 8-17.
83. Skarsgard, E.D.; Huang, L.; Reebye, S.C.; Yeung, A.Y.; Jia, W.W. Lentiviral vector-mediated, in vivo gene transfer to the tracheobronchial tree in fetal rabbits. J. Pediatr. Surg. 2005, 40, 1817-1821.
84. Tarantal, A.F.; McDonald, R.J.; Jimenez, D.F.; Lee, C.C.; O'Shea, C.E.; Leapley, A.C.; Won, R.H.; Plopper, C.G.; Lutzko, C.; Kohn, D.B. Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery. Mol. Ther. 2005, 12, 87-98.
85. Buckley, S.M.; Howe, S.J.; Sheard, V.; Ward, N.J.; Coutelle, C.; Thrasher, A.J.; Waddington, S.N.; McKay, T.R. Lentiviral transduction of the murine lung provides efficient pseudotype and developmental stage-dependent cell-specific transgene expression. Gene Ther. 2008, 15, 1167-1175.
86. Hughes, C.; Galea-Lauri, J.; Farzaneh, F.; Darling, D. Streptavidin paramagnetic particles provide a choice of three affinity-based capture and magnetic concentration strategies for retroviral vectors. Mol. Ther. 2001, 3, 623-630.
87. Tai, M.-F.; Chi, K.-M.; Lau, K.-H. W.; Baylink, D.J.; Chen, S.-T. Generation of magnetic retroviral vectors with magnetic nanoparticles. Rev. Adv. Mater. Sci. 2003, 5, 319-323.
88. Grubb, B.R.; Boucher, R.C. Pathophysiology of gene-targeted mouse models for cystic fibrosis. Physiol. Rev. 1999, 79, S193-S214.
89. Sun, X.; Yan, Z.; Yi, Y.; Li, Z.; Lei, D.; Rogers, C.S.; Chen, J.; Zhang, Y.; Welsh, M.J.; Leno, G.H.; Engelhardt, J.F. Adeno-associated virus-targeted disruption of the CFTR gene in cloned ferrets. J. Clin. Invest. 2008, 118, 1578-1583.
90. Rogers, C.S.; Hao, Y.; Rokhlina, T.; Samuel, M.; Stoltz, D.A.; Li, Y.; Petroff, E.; Vermeer, D.W.; Kabel, A.C.; Yan, Z.; Spate, L.; Wax, D.; Murphy, C.N.; Rieke, A.; Whitworth, K.; Linville, M.L.; Korte, S.W.; Engelhardt, J.F.; Welsh, M.J.; Prather, R.S. Production of CFTR-null and CFTR-DeltaF508 heterozygous pigs by adeno-associated virus-mediated gene targeting and somatic cell nuclear transfer. J. Clin. Invest. 2008, 118, 1571-1577.