Stem cell gene therapy, position effects and chromatin insulators

Stem Cells

Volumn 15, Issue SUPPL. 1, 1997, Pages 265-271

  Neff, Tobias ^b   Shotkoski, Frank ^a   Stamatoyannopoulos, George ^a  

^a UNIVERSITY OF WASHINGTON   (United States)

^b GOETHE UNIVERSITY   (Germany)

Author keywords

Chromatin insulators; Gene therapy; Globin; Position effects; Stem cells; Viral vectors

Indexed keywords

ANIMAL CELL; BINDING SITE; CHROMATIN; CONFERENCE PAPER; DROSOPHILA; GENE THERAPY; GENE TRANSFER; MOLECULAR CLONING; NONHUMAN; PROTEIN BINDING; SIGNAL TRANSDUCTION; STEM CELL; TRANSGENE;

ANIMALS; CHROMATIN; DNA-BINDING PROTEINS; DROSOPHILA; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEMATOPOIETIC STEM CELLS; HUMANS;

ANIMALIA;

EID: 0030667648     PISSN: 10665099     EISSN: None     Source Type: Journal    
DOI: 10.1002/stem.5530150834     Document Type: Article

References (32)

1. Wolffe AP. Insulating chromatin. Curr Biol 1994;4:85-87.
2. Stamatoyannopoulos G, Nienhuis AW. Hemoglobin switching. In: Stamatoyannopoulos G, Nienhuis AW, Majerus PW et al., eds. The Molecular Basis of Blood Diseases. 2nd ed. Philadelphia: W.B. Saunders, 1994:107-155.
3. Dunbar CE, Seidel N, Doren S et al. Improved retroviral gene transfer into murine and Rhesus peripheral blood or bone marrow repopulating cells primed in vivo with stem cell factor and granulocyte colony-stimulating factor. Proc Natl Acad Sci USA 1996;93:11871-11876.
4. Platt OS, Thorington BD, Brambilla DJ et al. Pain in sickle cell disease: rates and risk factors. N Engl J Med 1991;325:11-16.
5. Grosveld F, van Assendelft GB, Greaves DR et al. Position-independent, high-level expression of the human beta-globin gene in transgenic mice. Cell 1987;51:975-985.
6. Bender MA, Miller AD, Gelinas RE. Expression of the human beta-globin gene after retroviral transfer into murine erythroleukemia cells and human BFU-E cells. Mol Cell Biol 1988;8:1725-1735.
7. Novak U, Harris EA, Forrester W et al. High level beta-globin expression after retroviral transfer of locus activation region-containing human beta-globin gene derivatives into murine erythroleukemia cells. Proc Natl Acad Sci USA 1990;87:3386-3390.
8. Leboulch P, Huang GM, Humphries RK et al. Mutagenesis of retroviral vectors transducing human beta-globin gene and beta-globin locus control region derivatives results in stable transmission of an active transcriptional structure. EMBO J 1994;13:3065-3076.
9. Sadelain M, Wang CHJ, Antoniou M et al. Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene. Proc Natl Acad Sci USA 1995;92:6728-6732.
10. Ren S, Wong BY, Li J et al. Production of genetically stable high-titer retroviral vectors that carry a human gamma-globin gene under the control of the alpha-globin locus control region. Blood 1996;87:2518-2524.
11. Plavec I, Papayannopoulou T, Maury C et al. A human beta-globin gene fused to the human beta-globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus-transduced hematopoietic stem cells. Blood 1993;81:1384-1392.
12. Weinthal J, Nolta JA, Yu XJ et al. Expression of human glucocerebrosidase following retroviral vector-mediated transduction of murine hematopoietic stem cells. Bone Marrow Transplant 1991;8:403-412.
13. Challita PM, 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 1994;91:2567-2571.
14. Lim B, Williams DA, Orkin SH. Retrovirus-mediated gene transfer of human adenosine deaminase: expression of functional enzyme in murine hematopoietic stem cells in vivo. Mol Cell Biol 1987;7:3459-3465.
15. Nolta JA, Sender LS, Barranger JA et al. Expression of human glucocerebrosidase in murine long-term bone marrow cultures after retroviral vector-mediated transfer. Blood 1990;75:787-797.
16. Mitani K, Wakamiya M, Caskey CT. Long-term expression of retroviral-transduced adenosine deaminase in human primitive hematopoietic progenitors. Hum Gene Ther 1993;4:9-16.
17. Fink JK, Correl PH, Perry LK et al. Correction of glucocerebrosidase deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with Gaucher disease. Proc Natl Acad Sci USA 1990;87:2334-2338.
18. Hughes PF, Thacker JD, Hogge D et al. Retroviral gene transfer to primitive normal and leukemic hematopoietic cells using clinically applicable procedures. J Clin Invest 1992;89:1817-1824.
19. Bregni M, Magni M, Siena S et al. Human peripheral blood progenitors are optimal targets of retroviral-mediated gene transfer. Blood 1992;80:1418-1422.
20. + hematopoietic stem/ progenitor cells from human umbilical cord blood. J Exp Med 1993;178:2089-2096.
21. + cells purified from peripheral blood of breast cancer patients primed with chemotherapy and granulocyte-macrophage colony-stimulating factor. Hum Gene Ther 1994;5:203-208.
22. Cai H, Levine M. Modulation of enhancer-promoter interactions by insulators in the Drosophila embryo. Nature 1995;376:533-536.
23. Geyer PK, Corces VG. DNA position-specific repression of transcription by a Drosophila zinc finger protein. Genes Dev 1992;6:1865-1873.
24. Kellum R, Schedl P. A position-effect assay for boundaries of higher order chromosomal domains. Cell 1991;64:941-950.
25. Zhao K, Hart CM, Laemmli UK. Visualization of chromosomal domains with boundary element-associated factor (BEAF)-32. Cell 1995;81:879-889.
26. Chung JH, Whiteley M, Felsenfeld G. A 5′ element of the chicken β-globin domain serves as an insulator in human erythroid cells and protects against position effect in Drosophila. Cell 1993;74:505-514.
27. Li Q, Stamatoyannopoulos G. Hypersensitive site 5 of the human β-locus control region functions as a chromatin insulator. Blood 1994;84:1399-1401.
28. Zafarana G, Raguz S, Pruzina S et al. The regulation of β-globin gene expression: the analysis of hypersensitive site 5 (HS5) in the LCR. In: Stamatoyannopoulos G, ed. Proceedings of the 9th Conference on Hemoglobin Switching. Vol.1., Andover, UK: Intercept, 1995:39-44.
29. Stief A, Winter DM, Stratling WH et al. A nuclear DNA attachment element mediates elevated and position-independent gene activity. Nature 1989;341:343-345.
30. Zhong X-P, Krangel MS. An enhancer-blocking element between α and δ gene segments within the human T cell receptor α/δ locus. Proc Natl Acad Sci USA. 1997;94:5219-5224.
31. Hagstrom K, Muller M, Schedl P. Fab-7 functions as a chromatin domain boundary to ensure proper segment specification by the Drosophila bithorax complex. Genes Dev 1996;10:3202-3215.
32. Zhou J, Barolo S, Szymanski P et al. The Fab-7 element of the bithorax complex attenuates enhancer-promoter interactions in the Drosophila embryo. Genes Dev 1996;10:3195-3201.