Phase I/II study of intrapleural administration of a Serotype rh.10 replication-deficient adeno-associated virus gene transfer vector expressing the human α1-Antitrypsin cDNA to individuals with α1-Antitrypsin deficiency

Human Gene Therapy Clinical Development

Volumn 25, Issue 3, 2014, Pages 112-133

  Chiuchiolo, Maria J ^a   Kaminsky, Stephen M ^a   Sondhi, Dolan ^a   Mancenido, Denesy ^a   Hollmann, Charleen ^a   Crystal, Ronald G ^a  

^a WEILL CORNELL MEDICAL COLLEGE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA 1 ANTITRYPSIN; COMPLEMENTARY DNA;

ADENO ASSOCIATED VIRUS; ALPHA 1 ANTITRYPSIN DEFICIENCY; ARTICLE; DRUG EFFICACY; DRUG SAFETY; EPITHELIUM; FOOD AND DRUG ADMINISTRATION; GENE EXPRESSION; GENE REPLICATION; GENE TRANSFER; HUMAN; LUNG; LUNG FLUID; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PROTEIN BLOOD LEVEL; SEROTYPE; UNITED STATES; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; ALPHA 1-ANTITRYPSIN DEFICIENCY; CLINICAL TRIAL; COHORT ANALYSIS; DEPENDOPARVOVIRUS; FEMALE; FOLLOW UP; GENE THERAPY; GENE VECTOR; GENETICS; MALE; METABOLISM; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PROCEDURES;

ALPHA 1-ANTITRYPSIN; ALPHA 1-ANTITRYPSIN DEFICIENCY; COHORT STUDIES; DEPENDOVIRUS; DNA, COMPLEMENTARY; FEMALE; FOLLOW-UP STUDIES; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MALE; SEROGROUP;

EID: 84907259251     PISSN: 23248637     EISSN: 23248645     Source Type: Journal    
DOI: 10.1089/humc.2014.2513     Document Type: Article

References (108)

1. Alino, S.F. (1997). Long-term expression of the human alpha1-antitrypsin gene in mice employing anionic and cationic liposome vector. Biochem. Pharmacol. 54, 9-13.
2. Alino, S.F., Bobadilla, M., Garcia-Sanz, M., et al. (1993). In vivo delivery of human alpha 1-antitrypsin gene to mouse hepatocytes by liposomes. Biochem. Biophys. Res. Commun. 192, 174-181.
3. Alino, S.F., Crespo, J., Bobadilla, M., et al. (1994). Expression of human alpha 1-antitrypsin in mouse after in vivo gene transfer to hepatocytes by small liposomes. Biochem. Biophys. Res. Commun. 204, 1023-1030.
4. Alpha-1 Registry. (1998). Survival and FEV1 decline in individuals with severe deficiency of alpha1-antitrypsin. The Alpha-1-Antitrypsin Deficiency Registry Study Group. Am. J. Respir. Crit. Care Med. 158, 49-59.
5. Armstrong, M., Mark, L.J., Snyder, D.S., Parker, S.D. (1997). Safety of direct laryngoscopy as an outpatient procedure. Laryngoscope 107, 1060-1065.
6. Arruda, V.R., Schuettrumpf, J., Herzog, R.W., et al. (2004). Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adenoassociated viral vector serotype 1. Blood 103, 85-92.
7. ATS/ERS Task Force. (2003). American Thoracic Society/European Respiratory Society statement: standards for the diagnosis and management of individuals with alpha-1 antitrypsin deficiency. Am. J. Respir. Crit. Care Med. 168, 818-900.
8. Bao, J.J., Sifers, R.N., Kidd, V.J., et al. (1987). Molecular evolution of serpins: homologous structure of the human alpha 1-antichymotrypsin and alpha 1-antitrypsin genes. Biochemistry 26, 7755-7759.
9. Birrer, P., McElvaney, N.G., Chang-Stroman, L.M., and Crystal, R.G. (1991). Alpha 1-antitrypsin deficiency and liver disease. J. Inherit. Metab. Dis. 14, 512-525.
10. Brantly, M., Nukiwa, T., and Crystal, R.G. (1988). Molecular basis of alpha-1-antitrypsin deficiency. Am. J. Med. 84, 13-31.
11. Brantly, M.L., Wittes, J.T., Vogelmeier, C.F., et al. (1991). Use of a highly purified alpha 1-antitrypsin standard to establish ranges for the common normal and deficient alpha 1-antitrypsin phenotypes. Chest 100, 703-708.
12. Brantly, M.L., Spencer, L.T., Humphries, M., et al. (2006). Phase I trial of intramuscular injec-tion of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults. Hum. Gene Ther. 17, 1177-1186.
13. Brantly, M.L., Chulay, J.D., Wang, L., et al. (2009). Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc. Natl. Acad. Sci. USA 106, 16363-16368.
14. Brigham, K.L., Lane, K.B., Meyrick, B., et al. (2000). Transfection of nasal mucosa with a normal alpha1-antitrypsin gene in alpha1-antitrypsin-deficient subjects: comparison with protein therapy. Hum. Gene Ther. 11, 1023-1032.
15. Brody, S.L., Metzger, M., Danel, C., et al. (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.
16. Canonico, A.E., Conary, J.T., Meyrick, B.O., and Brigham, K.L. (1994a). Aerosol and intravenous transfection of human alpha 1-antitrypsin gene to lungs of rabbits. Am. J. Respir. Cell Mol. Biol. 10, 24-29.
17. Canonico, A.E., Plitman, J.D., Conary, J.T., et al. (1994b). No lung toxicity after repeated aerosol or intravenous delivery of plasmid-cationic liposome complexes. J. Appl. Physiol. 77, 415-419.
18. Canonico, A.E., Brigham, K.L., Carmichael, L.C., et al. (1996). Plasmid-liposome transfer of the alpha 1 antitrypsin gene to cystic fibrosis bronchial epithelial cells prevents elastaseinduced cell detachment and cytokine release. Am. J. Respir. Cell Mol. Biol. 14, 348-355.
19. Carrell, R.W., and Lomas, D.A. (2002). Alpha1-antitrypsin deficiency-a model for conformational diseases. N. Engl. J. Med. 346, 45-53.
20. Chiuchiolo, M.J., Kaminsky, S.M., Sondhi, D., et al. (2013). Intrapleural administration of an AAVrh.10 vector coding for human a1-antitrypsin for the treatment of a1-antitrypsin deficiency. Hum. Gene Ther. Clin. Dev. 24, 161-173.
21. Chulay, J.D., Ye, G.J., Thomas, D.L., et al. (2011). Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method. Hum. Gene Ther. 22, 155-165.
22. Cohen, A.B., Woo, S., Kelley, R., et al. (1988). Molecular genetics in pulmonary emphysema. Am. Rev. Respir. Dis. 138, 1041-1043.
23. Cox, D.W.,Woo, S.L., andMansfield, T. (1985). DNA restriction fragments associated with alpha 1-antitrypsin indicate a single origin for deficiency allele PI Z. Nature 316, 79-81.
24. Crepso, J., Blaya, C., Crespo, A., and Alino, S.F. (1996). Longterm expression of the human alpha1-antitrypsin gene in mice employing anionic and cationic liposome vectors. Biochem. Pharmacol. 51, 1309-1314.
25. Crystal, R.G. (1989). The alpha 1-antitrypsin gene and its deficiency states. Trends Genet. 5, 411-417.
26. Crystal, R.G. (1990). Alpha 1-antitrypsin deficiency, emphysema, and liver disease. Genetic basis and strategies for therapy. J. Clin. Invest. 85, 1343-1352.
27. Crystal, R.G. (1991). Alpha 1-antitrypsin deficiency: pathogenesis and treatment. Hosp. Pract. (Off Ed.) 26, 81-93.
28. Crystal, R.G., Brantly, M.L., Hubbard, R.C., et al. (1989). The alpha 1-antitrypsin gene and its mutations. Clinical consequences and strategies for therapy. Chest 95, 196-208.
29. Curiel, D.T., Vogelmeier, C., Hubbard, R.C., et al. (1990). Molecular basis of alpha 1-antitrypsin deficiency and emphysema associated with the alpha 1-antitrypsin Mmineral springs allele. Mol. Cell Biol. 10, 47-56.
30. De, B., Heguy, A., Leopold, P.L., et al. (2004a). Intrapleural administration of a serotype 5 adeno-associated virus coding for alpha1-antitrypsin mediates persistent, high lung and serum levels of alpha1-antitrypsin. Mol. Ther. 10, 1003-1010.
31. De, S.A., Vanderlinden, E., Demanet, C., et al. (2004b). Characterisation of pleural inflammation occurring after primary spontaneous pneumothorax. Eur. Respir. J. 23, 896-900.
32. De, B.P., Heguy, A., Hackett, N.R., et al. (2006). High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adenoassociated viruses. Mol. Ther. 13, 67-76.
33. de Serres, F.J. (2002). Worldwide racial and ethnic distribution of alpha1-antitrypsin deficiency: summary of an analysis of published genetic epidemiologic surveys. Chest 122, 1818-1829.
34. Dirksen, A., Dijkman, J.H., Madsen, F., et al. (1999). A randomized clinical trial of alpha(1)-antitrypsin augmentation therapy. Am. J. Respir. Crit. Care Med. 160, 1468-1472.
35. Ferrari, S., Geddes, D.M., and Alton, E.W. (2002). Barriers to and new approaches for gene therapy and gene delivery in cystic fibrosis. Adv. Drug Deliv. Rev. 54, 1373-1393.
36. Ferrari, S., Griesenbach, U., Geddes, D.M., and Alton E. (2003). Immunological hurdles to lung gene therapy. Clin. Exp. Immunol. 132, 1-8.
37. Flotte, T.R., and Mueller, C. (2011). Gene therapy for alpha-1 antitrypsin deficiency. Hum. Mol. Genet. 20, R87-R92.
38. Flotte, T.R., Trapnell, B.C., Humphries, M., et al. (2011). Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing a1-antitrypsin: interim results. Hum. Gene Ther. 22, 1239-1247.
39. Gadek, J., and Crystal, R.G. (1982). Alpha-1-antitrypsin deficiency. In Metabolic Basis of Inherited Disease. J.B. Stanbury, J.B. Wyngaarden, D.S. Frederickson, et al., eds. (McGrawHill, New York, NY) pp. 1450-1467.
40. Gadek, J.E., Fells, G.A., Zimmerman, R.L., et al. (1981). Antielastases of the human alveolar structures. Implications for the protease-antiprotease theory of emphysema. J. Clin. Invest. 68, 889-898.
41. Gao, G., Vandenberghe, L.H., and Wilson, J.M. (2005). New recombinant serotypes of AAV. Curr. Gene Ther. 5, 285-297.
42. Garver, R.I., Jr., Chytil, A., Courtney, M., and Crystal, R.G. (1987). Clonal gene therapy: in vivo expression of a transplanted monoclonal population of murine fibroblasts containing a retrovirus inserted human alpha 1-antitrypsin gene. Trans. Assoc. Am. Phys. 100, 10-20.
43. Greene, C.M., Miller, S.D., Carroll, T., et al. (2008). Alpha-1 antitrypsin deficiency: a conformational disease associated with lung and liver manifestations. J. Inherit. Metab. Dis. 31, 21-34.
44. Harvey, B.G., Hackett, N.R., El Sawy, T., et al. (1999a). Variability of human systemic humoral immune responses to adenovirus gene transfer vectors administered to different organs. J. Virol. 73, 6729-6742.
45. Harvey, B.G., Leopold, P.L., Hackett, N.R., et al. (1999b). Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirus. J. Clin. Invest. 104, 1245-1255.
46. Harvey, B.G., Hackett, N.R., Ely, S., and Crystal, R.G. (2001). Host responses and persistence of vector genome following intrabronchial administration of an E1(-)E3(-) adenovirus gene transfer vector to normal individuals. Mol. Ther. 3, 206-215.
47. High, K. (2002a). AAV-mediated gene transfer for hemophilia. Genet. Med. 4, 56S-61S.
48. High, K.A. (2002b). Adeno-associated virus-mediated gene transfer for hemophilia B. Int. J. Hematol. 76, 310-318.
49. High, K.A. (2004). Clinical gene transfer studies for hemophilia B. Semin. Thromb. Hemost. 30, 257-267.
50. Hubbard, R.C., and Crystal, R.G. (1990). Augmentation therapy of alpha 1-antitrypsin deficiency. Eur. Respir. J. Suppl. 9, 44s-52s.
51. Hubbard, R.C., Sellers, S., Czerski, D., et al. (1988). Biochemical efficacy and safety of monthly augmentation therapy for alpha 1-antitrypsin deficiency. JAMA 260, 1259-1264.
52. Jaffe, H.A., Danel, C., Longenecker, G., et al. (1992). Adenovirus-mediated in vivo gene transfer and expression in normal rat liver. Nat. Genet. 1, 372-378.
53. Jones, E.A., Vergalla, J., Steer, C.J., et al. (1978). Metabolism of intact and desialylated alpha 1-antitrypsin. Clin. Sci. Mol. Med. 55, 139-148.
54. Katzenstein, A.L., Bloor, C.M., and Leibow, A.A. (1976). Diffuse alveolar damage-the role of oxygen, shock, and related factors. A review. Am. J. Pathol. 85, 209-228.
55. Katzenstein, A.L., Myers, J.L., and Mazur, M.T. (1986). Acute interstitial pneumonia. A clinicopathologic, ultrastructural, and cell kinetic study. Am. J. Surg. Pathol. 10, 256-267.
56. Kay, M.A., Baley, P., Rothenberg, S., et al. (1992). Expression of human alpha 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc. Natl. Acad. Sci. USA 89, 89-93.
57. Larson, P.J., and High, K.A. (2001). Gene therapy for hemophilia B: AAV-mediated transfer of the gene for coagulation factor IX to human muscle. Adv. Exp. Med. Biol. 489, 45-57.
58. Laurell, C.B., and Eriksson, S.E. (1963). The electrophoretic alpha-globulin pattern of serum in alpha-antitrypsin deficiency. Scand. J. Clin. Invest. 15, 132-140.
59. Levy, M.Y., Barron, L.G., Meyer, K.B., and Szoka, F.C., Jr. (1996). Characterization of plasmid DNA transfer into mouse skeletal muscle: evaluation of uptake mechanism, expression and secretion of gene products into blood. Gene Ther. 3, 201-211.
60. Light, R.W. (2001). Physiology of the pleural space. In Pleural Diseases. R.W. Light, ed. (Lippincott Williams & Wilkins, Philadelphia, PA) pp. 8-20.
61. Limberis, M.P., Vandenberghe, L.H., Zhang, L., et al. (2009). Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro. Mol. Ther. 17, 294-301.
62. Liu, Y.L., Mingozzi, F., Rodriguez-Colon, S.M., et al. (2004). Therapeutic levels of factor IX expression using a musclespecific promoter and adeno-associated virus serotype 1 vector. Hum. Gene Ther. 15, 783-792.
63. Loebermann, H., Tokuoka, R., Deisenhofer, J., and Huber, R. (1984). Human alpha 1-proteinase inhibitor. Crystal structure analysis of two crystal modifications, molecular model and preliminary analysis of the implications for function. J. Mol. Biol. 177, 531-557.
64. Lomas, D.A., and Parfrey, H. (2004). Alpha1-antitrypsin deficiency. 4: molecular pathophysiology. Thorax 59, 529-535.
65. Lomas, D.A., Evans, D.L., Finch, J.T., et al. (1992). The mechanism of Z alpha 1-antitrypsin accumulation in the liver. Nature 357, 605-607.
66. Long, G.L., Chandra, T., Woo, S.L., et al. (1984). Complete sequence of the cDNA for human alpha 1-antitrypsin and the gene for the S variant. Biochemistry 23, 4828-4837.
67. Lu, Y., Campbell-Thompson, M., Li, C., et al. (2004) Functional human alpha 1 antitrypsin secreted from muscle transduced by adeno-associated virus (rAAV1) vector. Mol. Ther. 9, S41-S42.
68. Lu, Y., Choi, Y.K., Campbell-Thompson, M., et al. (2006). Therapeutic level of functional hu-man alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adenoassociated virus (rAAV1) vector. J. Gene Med. 8, 730-735.
69. Luisetti, M., Miravitlles, M., and Stockley, R.A. (2002). Alpha l-antitrypsin deficiency: a report from the 2nd meeting of the Alpha One International Registry, Rapallo (Genoa, Italy), 2001. Eur. Respir. J. 20, 1050-1056.
70. Mae, M., and Crystal, R.G. (2002). Gene transfer to the pleural mesothelium as a strategy to deliver proteins to the lung parenchyma. Hum. Gene Ther. 13, 1471-1482.
71. Manno, C.S., Chew, A.J., Hutchison, S., et al. (2003). AAVmediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101, 2963-2972.
72. Mastrangeli, A., Harvey, B.G., and Yao, J. (1996). "Seroswitch" adenovirus-mediated in vivo gene transfer: circumvention of anti-adenovirus humoral immune defenses against repeat adenovirus vector administration by changing the adenovirus serotype. Hum. Gene Ther. 7, 79-87.
73. Matot, I., Kramer, M.R. (2000). Sedation in outpatient bronchoscopy. Respir. Med. 94, 1145-1153.
74. Momex, J.F., Chytil-Weir, A., Martinet, Y., et al. (1986). Expression of the alpha-1-antitrypsin gene in mononuclear phagocytes of normal and alpha-1-antitrypsin-deficient individuals. J. Clin. Invest. 77, 1952-1961.
75. Mueller, C., and Flotte, T.R. (2013). Gene-based therapy for alpha-1 antitrypsin deficiency. COPD 10 Suppl 1, 44-49.
76. Mullins, C.D., Huang, X., Merchant, S., and Stoller, J.K. (2001). The direct medical costs of alpha(l)-antitrypsin deficiency. Chest 119, 745-752.
77. Myers, J.L., and Katzenstein, A.L. (1988). Ultrastructural evidence of alveolar epithelial injury in idiopathic bronchiolitis obliterans-organizing pneumonia. Am. J. Pathol. 132, 102-109.
78. Ogushi, F., Fells, G.A., Hubbard, R.C., et al. (1987). Z-type alpha 1-antitrypsin is less competent than M1-type alpha 1-antitrypsin as an inhibitor of neutrophil elastase. J. Clin. Invest. 80, 1366-1374.
79. Ogushi, F., Hubbard, R.C., Vogelmeier, C., et al. (1991). Risk factors for emphysema. Cigarette smoking is associated with a reduction in the association rate constant of lung alpha 1-antitrypsin for neutrophil elastase. J. Clin. Invest. 87, 1060-1065.
80. Pertmer, T.M., Eisenbraun, M.D., McCabe, D., et al. (1995). Gene gun-based nucleic acid immunization: elicitation of humoral and cytotoxic T lymphocyte responses following epidermal delivery of nanogram quantities of DNA. Vaccine 13, 1427-1430.
81. Qiu, P., Ziegelhoffer, P., Sun, J., and Yang, N.S. (1996). Gene gun delivery of mRNA in situ results in efficient transgene expression and genetic immunization. Gene Ther. 3, 262-268.
82. Rennard, S.I., Basset, G., Lecossier, D., et al. (1986). Estimation of volume of epithelial lining fluid recovered by lavage using urea as marker of dilution. J. Appl. Physiol. 60, 532-538.
83. Rosenfeld, M.A., Siegfried, W., Yoshimura, K., et al. (1991). Adenovirus-mediated transfer of a recombinant alpha 1-antitrypsin gene to the lung epithelium in vivo. Science 252, 431-434.
84. Rosenfeld, M.A., Yoshimura, K., Trapnell, B.C., et al. (1992). In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium. Cell 68, 143-155.
85. Rosenfeld, M.A., Ronald, G., and Crystal, R.G. (1993). Gene therapy for pulmonary diseases. Pathol. Biol. (Paris) 41, 677-680.
86. Schreier, H., Gagne, L., Bock, T., et al. (1997). Physicochemical properties and in vitro toxicity of cationic liposome cDNA complexes. Pharm. Acta Helv. 72, 215-223.
87. Schroeder, W.T., Miller, M.F., Woo, S.L., and Saunders, G.F. (1985). Chromosomal localization of the human alpha 1-antitrypsin gene (PI) to 14q31-32. Am. J. Hum. Genet. 37, 868-872.
88. Seixas, S., Garcia, O., Trovoada, M.J., et al. (2001). Patterns of haplotype diversity within the serpin gene cluster at 14q32.1: insights into the natural history of the alpha1-antitrypsin polymorphism. Hum. Genet. 108, 20-30.
89. Setoguchi, Y., Jaffe, H.A., Chu, C.S., and Crystal, R.G. (1994). Intraperitoneal in vivo gene therapy to deliver alpha 1-antitrypsin to the systemic circulation. Am. J. Respir. Cell Mol. Biol. 10, 369-377.
90. Silverman, E.K., and Sandhaus, R.A. (2009). Clinical practice. Alpha1-antitrypsin deficiency. N. Engl. J.Med. 360, 2749-2757.
91. Song, S., Morgan, M., Ellis, T., et al. (1998) Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc. Natl. Acad. Sci. USA 95, 14384-14388.
92. Song, S., Embury, J., Laipis, P.J., et al. (2001). Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors. Gene Ther. 8, 1299-1306.
93. Song, S., Scott-Jorgensen, M., Wang, J., et al. (2002). Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspects. Mol. Ther. 6, 329-335.
94. Stecenko, A.A., and Brigham, K.L. (2003). Gene therapy progress and prospects: alpha-1 antitrypsin. Gene Ther. 10, 95-99.
95. Stockley, R.A. (2000). Alpha-1-antitrypsin deficiency: what next? Thorax 55, 614-618.
96. Stocks, J.M., Brantly, M.L., Wang-Smith, L., et al. (2010). Pharmacokinetic comparability of Prolastin-C to Prolastin-in alpha-antitrypsin deficiency: a randomized study. BMC Clin. Pharmacol. 30, 10-13.
97. Stoller, J.K., and Aboussouan, L.S. (2005). Alpha1-antitrypsin deficiency. Lancet 365, 2225-2236.
98. Stoller, J.K., and Aboussouan, L.S. (2012). A review of alpha1-antitrypsin deficiency. Am. J. Respir. Crit. Care Med. 185, 246-259.
99. Strange, P. (2001). Pleural diseases. In Egan's Fundamentals of Respiratory Care. C.L. Scanlan, R.L. Wilkins, and J.K. Stoller, eds. (Elsevier Inc., St. Louis, MO) pp. 475-490.
100. Teramoto, S., Bartlett, J.S., McCarty, D., et al. (1998). Factors influencing adeno-associated virus-mediated gene transfer to human cystic fibrosis airway epithelial cells: comparison with adenovirus vectors. J. Virol. 72, 8904-8912.
101. Thompson Healthcare (2010). Red Book: Pharmacy's Fundamental Reference. (Thomson Reuters, PDR Network, New York).
102. Tuder, R.M., Janciauskiene, S.M., and Petrache, I. (2010). Lung disease associated with alpha1-antitrypsin deficiency. Proc. Am. Thorac. Soc. 7, 381-386.
103. Vadolas, J., Williamson, R., and Ioannou, P.A. (2002). Gene therapy for inherited lung disorders: an insight into pulmonary defence. Pulm. Pharmacol. Ther. 15, 61-72.
104. Virella-Lowell, I., Poirier, A., Chesnut, K.A., et al. (2000). Inhibition of recombinant adeno-associated virus (rAAV) transduction by bronchial secretions from cystic fibrosis patients. Gene Ther. 7, 1783-1789.
105. Wewers, M.D., Casolaro, M.A., and Crystal, R.G. (1987). Comparison of alpha-1-antitrypsin levels and antineutrophil elastase capacity of blood and lung in a patient with the alpha-1-antitrypsin phenotype null-null before and during alpha-1-antitrypsin augmentation therapy. Am. Rev. Respir. Dis. 135, 539-543.
106. Wu, S.S., de Chadarevian, J.P., McPhaul, L., et al. (2002).Coexpression and accumulation of ubiquitin + 1 and ZZ proteins in livers of children with alpha-1 antitrypsin deficiency. Pediatr. Dev. Pathol. 5, 293-298.
107. Yang, Y., Li, Q., Ertl, H.C., et al. (1995). Cellular and humoral immune responses to viral anti-gens create barriers to lungdirected gene therapy with recombinant adenoviruses. J. Virol. 69, 2004-2015.
108. Zamora, N.P., Pla, R.V., Del Rio, P.G., et al. (2008). Intravenous human plasma-derived augmentation therapy in alpha 1-antitrypsin deficiency: from pharmacokinetic analysis to individualizing therapy. Ann. Pharmacother. 42, 640-646.