Expression of human alpha1 antitrypsin in murine hematopoietic cells in vivo after retrovirus-mediated gene transfer

Molecular Genetics and Metabolism

Volumn 63, Issue 3, 1998, Pages 198-204

  Saylors III, Robert L ^a   Wall, Donna A ^b  

^a ARKANSAS CHILDREN S HOSPITAL   (United States)

^b Cardinal Glennon Hospital   (United States)

Author keywords

Alpha1 antitrypsin deficiency; Bone marrow; Gene therapy; Retrovital vectors

Indexed keywords

ALPHA 1 ANTITRYPSIN; COMPLEMENTARY DNA; DIHYDROFOLATE REDUCTASE; DNA;

ALPHA 1 ANTITRYPSIN DEFICIENCY; ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; BONE MARROW TRANSPLANTATION; CONTROLLED STUDY; ENCEPHALOMYOCARDITIS VIRUS; EXPRESSION VECTOR; GENE TRANSFER; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL; MALE; MOLONEY LEUKEMIA ONCOVIRUS; MOUSE; NONHUMAN; POLYMERASE CHAIN REACTION; PRIORITY JOURNAL; RETROVIRUS; SOUTHERN BLOTTING;

ANIMALIA; ENCEPHALOMYOCARDITIS VIRUS; MOLONEY MURINE LEUKEMIA VIRUS; MURINAE; ONCOVIRINAE; RODENTIA; UNIDENTIFIED RETROVIRUS;

EID: 0031695436     PISSN: 10967192     EISSN: None     Source Type: Journal    
DOI: 10.1006/mgme.1997.2665     Document Type: Article

References (29)

1. Cox DW. a1-antitrypsin deficiency. In The Metabolic and Molecular Basis of Inherited Disease (Scriver CR, Beaudet AL, Sly WS, Valle D, Eds.). New York: McGraw-Hill, 1995, 7th ed., pp 4125-4158.
2. Hubbard RC, Crystal RG. Alpha-1-antitrypsin augmentation therapy for alpha-1-antitrypsin deficiency. Am J Med 84(suppl. 6A):52-62, 1988.
3. Wewers MD, Casolaro MA, Sellers SE, Swayze SC, McPhaul KM, Wittes JT, Crystal RG. Replacement therapy for alphal-antitrypsin deficiency associated with emphysema. N Engl J Med 316:1055-1062, 1987.
4. Kay MA, Baley P, Rothenberg S, Leland F, Fleming L, Ponder KP, Liu T-J, Finegold M, Darlington G, Pokorny W, Woo SLC. Expression of human a1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc Natl Acad Sci USA 89:89-93, 1992.
5. Rettinger SD, Ponder KP, Saylors RL, Kennedy SC, Hafenrichter DG, Flye MW. In vivo hepatocyte transduction with retrovirus during in-flow occlusion. J Surg Res 54:418-425, 1993.
6. Rettinger SD, Kennedy SC, Wu X, Saylors RL, Hafenrichter DG, Flye MW, Ponder KP. Liver-directed gene therapy: Quantitative evaluation of promoter elements by using in vivo retroviral transduction. Proc Natl Acad Sci USA 91:1460-1464, 1994.
7. Williams DA, Orkin SH, Mulligan RC. Retrovirus-mediated transfer of human adenosine deaminase gene sequences into cells in culture and into murine hematopoietic cells in vivo. Proc Natl Acad Sci USA 83:2566-2570, 1986.
8. Osborne WRA, Hock RA, Kaleko M, Miller AD. Long-term expression of human adenosine deaminase in mice after transplantation of bone marrow infected with amphotrophic retroviral vectors. Hum Gene Ther 1:31-41, 1990.
9. van Beusechem VW, Kukler A, Heidt PJ, Valerio D. Long-term expression of human adenosine deaminase in rhesus monkeys transplanted with retrovirus-infected bone marrow cells. Proc Natl Acad Sci USA 89:7640-7644, 1992.
10. Fink JK, Correll PH, Perry LK, Brady RO, Karlsson S. Correction of glucocerebrosidase deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with Gaucher disease. Proc Natl Acad Sci USA 87:2334-2338, 1990.
11. Kohn DB, Nolta JA, Weinthal J, Bahner I, Yu XJ, Lilley J, Crooks GM. Toward gene therapy for Gaucher disease. Hum Gene Ther 2:101-105, 1991.
12. Markowitz D, Goff S, Bank A. A safe packaging line for gene transfer: Separating viral genes on two different plasmids. J Virol 62:1120-1124, 1988.
13. Bender MA, Palmer TD, Gelinas RD, Miller AD. Evidence that the packaging signal of moloney murine leukemia virus extends into the gag region. J Virol 61:1639-1646, 1987.
14. Simonsen CC, Levinson AD. Isolation and expression of an altered mouse dihydrofolate reductase cDNA. Proc Natl Acad Sci USA 80:2495-2499, 1983.
15. Ghattas IR, Sanes JR, Majors JE. The encephalomyocarditis virus internal ribosome entry site allows efficient coexpression of two genes from a recombinant provirus in cultured cells and in embryos. Mol Cell Biol 11:5848-5859, 1991.
16. Cornetta K, Morgan RA, Anderson WF. Safety issues related to retroviral-mediated gene transfer in humans. Hum Gene Ther 2:5-14, 1991.
17. Sambrook J, Fritsch EF, Maniatis T. Molecular Cloning -A Laboratory Manual. Cold Spring Harbor, NY: Cold Spring Harbor Press, 1989, 2nd ed.
18. Feinberg AP, Vogelstein B. A technique for radiolabeling DNA restriction endonuclease fragments to high specific activity. Anal Biochem 132:6-13, 1983.
19. Lubin MB, Elashoff JD, Wang S-J, Rotter JI, Toyoda H. Precise gene dosage determination by polymerase chain reaction: Theory, methodology, and statistical approach. Mol Cell Probes 5:307-317, 1991.
20. Williams DA. Expression of introduced genetic sequences in hematopoietic cells following retroviral-mediated gene transfer. Hum Gene Ther 1:229-239, 1990.
21. Corey CA, DeSilva AD, Holland CA, Williams DA. Serial transplantation of methotrexate-resistant bone marrow: Protection of murine recipients from drug toxicity by progeny of transduced stem cells. Blood 75:337-343, 1990.
22. Dick JE, Magli MC, Huszar D, Phillips RA, Bernstein A. Introduction of a selectable gene into primitive stem cells capable of long-term reconstitution of the hematopoietic system of W/Wv mice. Cell 42:71-79, 1985.
23. Keller G, Paige C, Gilboa E, Wagner EF. Expression of a foreign gene in myeloid and lymphoid cells derived from multipotent haematopoietic precursors. Nature 318:149-154, 1985.
24. Challita P, Kohn DB. 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 91:2567-2571, 1994.
25. Feinstein SC, Ross SR, Yamamoto K. Chromosome position effects determine transcriptional potential of integrated mammary tumor virus DNA. J Mol Biol 156:549-565, 1982.
26. Jolly DJ, Willis RC, Friedman T. Variable stability of a selectable provirus after retroviral vector gene transfer into human cells. Mol Cell Biol 6:1141-1147, 1986.
27. Correll PH, Colilla S, Karlsson S. Retroviral vector design for long-term expression in murine hematopoietic cells in vivo. Blood 84:1812-1822, 1994.
28. Ohashi T, Boggs S, Robbins P, Bahnson A, Patrene K, Wei F-S, Wei J-F, Li J, Lucht L, Fei Y, Clark S, Kimak M, He H, Mowery-Rushton P, Barranger JA. Efficient transfer and sustained high expression of the human glucocerebrosidase gene in mice and their functional macrophages following transplantation of bone marrow transduced by a retroviral vector. Proc Natl Acad Sci USA 89:11332-11336, 1992.
29. Riviere I, Brose K, Mulligan RC. Effects of retroviral vector design on expression of human adenosine deaminase in murine bone marrow transplant recipients engrafted with genetically modified cells. Proc Natl Acad Sci USA 92:6733-6737, 1995.