Gene therapy for α1-antitrypsin deficiency

Gene Therapy in Lung Disease

Volumn , Issue , 2002, Pages 407-418

  Flotte, Terence R ^a  

^a UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 84942824019     PISSN: None     EISSN: None     Source Type: Book    
DOI: None     Document Type: Chapter

References (39)

1. Garver RI, Chytil A, Courtney M, Crystal RG. Clonal gene therapy: transplanted mouse fibroblast clones express human alpha 1-antitrypsin gene in vivo. Science 1987; 237:762-464.
2. Garver RI, Chytil A, Courtney M, Crystal RG. 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 Physicians 1987; 100:10-20.
3. Kay MA, Li Q, Liu TJ, Leland F, Toman C, Finegold M, Woo SL. Hepatic gene therapy: persistent expression of human alpha 1-antitrypsin in mice after direct gene delivery in vivo. Hum Gene Ther 1992; 3:641-647.
4. Kay MA, Baley P, Rothenberg S, Leland F, Fleming L, Ponder KP, Liu T, Finegold M, Darlington G, Pokorny W, Woo SL. Expression of human alpha 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc Natl Acad Sci USA 1992; 89:89-93.
5. Rosenfeld MA, Siegfried W, Yoshimura K, Yoneyama K, Fukayama M, Stier LE, Paakko PK, Gilardi P, Stratford-Perricaudet LD, Perricaudet M, Crystal RG. Adenovirus-mediated transfer of A recombinant alpha 1-antitrypsin gene to the lung epithelium in vivo. Science 1991; 252:431-434.
6. Siegfried W, Rosenfeld M, Stier L, Stratford-Perricaudet L, Perricaudet M, Pavirani A, Lecocq JP, Crystal RG. Polarity of secretion of alpha 1-antitrypsin by human respiratory epithelial cells after adenoviral transfer of A human alpha 1-antitrypsin cDNA. Am J Respir Cell Mol Biol 1995; 12:379-384.
7. Setoguchi Y, Jaffe HA, Danel C, Crystal RG. Ex vivo and in vivo gene transfer to the skin using replication-deficient recombinant adenovirus vectors. J Invest Dermatol 1994; 102:415-421.
8. Setoguchi Y, Jaffe HA, Chu CS, Crystal RG. Intraperitoneal in vivo gene therapy to deliver alpha 1-antitrypsin to the systemic circulation. Am J Respir Cell Mol Biol 1994; 10:369-377.
9. Morral N, Parks RJ, Zhou H, Langston C, Schiedner G, Quinones J, Graham FL, Kochanek S, Beaudet AL. High doses of A helper-dependent adenoviral vector yield supraphysiological levels of alpha 1-antitrypsin with negligible toxicity. Hum Gene Ther 1998; 9:2709-2716.
10. Schiedner G, Morral N, Parks RJ, Wu Y, Koopmans SC, Langston C, Graham FL, Beaudet AL, Kochanek S. Genomic DNA transfer with A high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nat Genet 1998; 18:180-183.
11. Canonico AE, Plitman JD, Conary JT, Meyrick BO, Brigham KL. No lung toxicity after repeated aerosol or intravenous delivery of plasmid-cationic liposome complexes. J Appl Physiol 1994; 77:415-419.
12. Canonico AE, Conary JT, Meyrick BO, Brigham KL. Aerosol and intravenous transfection of human alpha 1-antitrypsin gene to lungs of rabbits. Am J Respir Cell Mol Biol 1994; 10:24-29.
13. Canonico AE, Brigham KL, Carmichael LC, Plitman JD, King GA, Blackwell TR, Christman JW. Plasmid-liposome transfer of the alpha 1-antitrypsin gene to cystic fibrosis bronchial epithelial cells prevents elastase-induced cell detachment and cytokine release. Am J Respir Cell Mol Biol 1996; 14:348-355.
14. Alino SF, Crespo J, Bobadilla M, Lejarreta M, Blaya C, Crespo A. Expression of human alpha 1-antitrypsin in mouse after in vivo gene transfer to hepatocytes by small liposomes. Biochem Biophys Res Commun 1994; 204:1023-1030.
15. Alino SF, Bobadilla M, Crespo J, Lejarreta M. Human alpha 1-antitrypsin gene transfer to in vivo mouse hepatocytes. Hum Gene Ther 1996; 7:531-536.
16. Berns KI, Linden RM. The cryptic life style of adeno-associated virus. Bioessays 1995; 17:237-245.
17. Blacklow NR, Hoggan MD, Kapikian AZ, Austin JB, Rowe WP. Epidemiology of adenovirus-associated virus infection in A nursery population. Am J Epidemiol 1968; 88:368-378.
18. Blacklow NR. Adeno-associated viruses of humans. In: Pattison JR, ed., Parvoviruses and Human Disease. Boca Raton, FL: CRC Press, 1988;165-174.
19. Grimm D, Kern A, Rittner K, Kleinschmidt JA. Novel tools for production and purification of recombinant adenoassociated virus vectors. Hum Gene Ther 1998; 9:2745-2760.
20. Zolotukhin S, Byrne BJ, Mason E, Zolotukhin I, Potter M, Chesnut K, Summerford C, Samulski RJ, Muzyczka N. Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther 1999; 6:973-985.
21. Flotte T, Carter B, Conrad C, Guggino W, Reynolds T, Rosenstein B, Taylor G, Walden S, Wetzel R. A phase I study of an adeno associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum Gene Ther 1996; 7:1145-1159.
22. Virella-Lowell I, Poirier A, Chesnut KA, Brantly M, Flotte TR. Inhibition of recombinant adeno-associated virus (rAAV) transduction by bronchial secretions from cystic fibrosis patients. Gene Ther 2000; 7:1783-1789.
23. Wagner JA, Reynolds T, Moran ML, Moss RB, Wine JJ, Flotte TR, Gardner P. Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus. Lancet 1998; 351:1702-1703.
24. Wagner JA, Moran ML, Messner AH, Daifuku R, Conrad CK, Reynolds T, Guggino WB, Moss RB, Carter BJ, Wine JJ, Flotte TR, Gardner P. A phase I/II study of tgAAV-CF for the treatment of chronic sinusitis in patients with cystic fibrosis. Hum Gene Ther 1998; 9:889-909.
25. Wagner JA, Nepomuceno IB, Messner AH, Moran MI, Batson EP, Desch JK, Nor- bash AM, Conrad CK, Guggino WB, Flotte TR, Wine JJ, Carter BJ, Moss RB, Gardner P. A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in CF patients with antrostomies. Human Gene Therapy 2002. In press.
26. Wagner JA, Messner AH, Moran ML, Daifuku R, Kouyama K, Desch JK, Manly S, Norbash AM, Conrad CK, Friborg S, Reynolds T, Guggino WB, Moss RB, Carter BJ, Wine JJ, Flotte TR, Gardner P. Safety and biological efficacy of an adeno- associated virus vector-cystic fibrosis transmembrane conductance regulator (AAV- CFTR) in the cystic fibrosis maxillary sinus. Laryngoscope 1999; 109:266-274.
27. Flotte TR, Afione SA, Conrad C, Mcgrath SA, Solow R, Oka H, Zeitlin PL, Guggino WB, Carter BJ. Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc Natl Acad Sci USA 1993; 90:10613-10617.
28. Flotte TR, Carter BJ. Adeno-associated virus vectors for gene therapy of cystic fibrosis. Methods Enzymol 1998; 292:717-732.
29. Song S, Morgan M, Ellis T, Poirier A, Chesnut K, Wang J, Brantly M, Muzyczka N, Byrne BJ, Atkinson M, Flotte TR. Sustained secretion of human alpha 1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc Natl Acad Sci USA 1998; 95:14384-14385.
30. Song S, Laipis PJ, Berns KI, Flotte TR. Effect of DNA-dependent protein kinase on the molecular fate of the rAAV2 genome in skeletal muscle. Proc Natl Acad Sci USA 2001; 98:4084-4088.
31. Song S, Laipis P, Embury J, Berns K, Crawford J, Flotte TR. Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors. Gene Ther 2001; 8:1299-1306.
32. Virella-Lowell I, Zusman B, Conlon T, Morgan M, Chesnut KA, Ferkol T, Flotte TR. A CMV/beta actin hybrid promoter enhances rAAV vector expression in the lung. Submitted.
33. Flotte TR, Afione SA, Zeitlin PL. Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration. Am J Respir Cell Mol Biol 1994; 11:517-521.
34. Afione SA, Conrad CK, Kearns WG, Chunduru S, Adams R, Reynolds TC, Guggino WB, Cutting GR, Carter BJ, Flotte TR. In vivo model of adeno-associated virus vector persistence and rescue. J Virol 1996; 70:3235-3241.
35. Kearns WG, Afione SA, Fulmer SB, Pang MC, Erikson D, Egan M, Landrum MJ, Flotte TR, Cutting GR. Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in A site-specific fashion in an immortalized epithelial cell line. Gene Ther 1996; 3:748-755.
36. Hernandez YJ, Wang J, Kearns WG, Loiler S, Poirier A, Flotte TR. Latent adeno- associated virus infection elicits humoral but not cell-mediated immune responses in A nonhuman primate model. J Virol 1999; 73:8549-8558.
37. Zern MA, Ozaki I, Duan L, Pomerantz R, Liu SL, Strayer DS. A novel SV40-based vector successfully transduces and expresses an alpha 1-antitrypsin ribozyme in A human hepatoma-derived cell line. Gene Ther 1999; 6:114-120.
38. Kren BT, Parashar B, Bandyopadhyay P, Chowdhury NR, Chowdhury JR, Steer CJ. Correction of the UDP-glucuronosyltransferase gene defect in the gunn rat model of Crigler-Najjar syndrome type I with A chimeric oligonucleotide. Proc Natl Acad Sci USA 1999; 96:10349-10354.
39. Kren BT, Cole-Strauss A, Kmiec EB, Steer CJ. Targeted nucleotide exchange in the alkaline phosphatase gene of HuH-7 cells mediated by A chimeric RNA/DNA oligonucleotide. Hepatology 1997; 25:1462-1468.