Differences of globin transgene expression in stably transfected cell lines and transgenic mice

Blood

Volumn 105, Issue 8, 2005, Pages 3346-3352

  Li, Qiliang ^a,b   Emery, David W ^a   Han, Hemei ^a   Sun, Jin ^a   Yu, Man ^a   Stamatoyannopoulos, George ^a  

^a UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF WASHINGTON   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA GLOBIN; DELTA GLOBIN; DEOXYRIBONUCLEASE; DNASE 1 HYPERSENSITIVE SITE 40; GAMMA GLOBIN; PROTEIN SUBUNIT; UNCLASSIFIED DRUG;

ANIMAL CELL; ARTICLE; CHROMATIN ASSEMBLY AND DISASSEMBLY; CHROMATIN STRUCTURE; CHROMOSOME POSITIONING; CONFORMATION; CONTROLLED STUDY; CORRELATION ANALYSIS; ENZYME ACTIVE SITE; ENZYME SUBUNIT; ERYTHROLEUKEMIA; GENE CASSETTE; GENE EXPRESSION; GENETIC TRANSFECTION; IMMUNOPRECIPITATION; MOLECULAR DYNAMICS; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; TRANSCRIPTION REGULATION; TRANSGENE; TRANSGENIC MOUSE;

EID: 17044383880     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood-2004-03-0987     Document Type: Article

References (34)

1. Higgs DR, Sharpe JA, Wood WG. Understanding alpha globin gene expression: a step towards effective gene therapy. Semin Hematol. 1998;35:93-104.
2. Sharpe JA, Chan-Thomas PS, Lida J, Ayyub H, Wood WG, Higgs DR. Analysis of the human alpha globin upstream regulatory element (HS-40) in transgenic mice. EMBO J. 1992;11:4565-4572.
3. Sharpe JA, Wells DJ, Whitelaw E, Vyas P, Higgs DR, Wood WG. Analysis of the human alpha-globin gene cluster in transgenic mice. Proc Natl Acad Sci U S A. 1993;90:11262-11266.
4. Higgs DR, Wood WG, Jarman AP, et al. A major positive regulatory region located far upstream of the human alpha-globin gene locus. Genes Dev. 1990;4:1588-1601.
5. Ren S, Wong BY, Li J, Luo XN, Wong PM, Atweh GF. Production of genetically stable high-liter retroviral vectors that carry a human gamma-globin gene under the control of the alpha-globin locus control region. Blood. 1996;87:2518-2524.
6. Li Q, Emery DW, Fernandez M, Han H, Stamatoyannopoulos G. Development of viral vectors for gene therapy of beta-chain hemoglobinopathies: optimization of a gamma-globin gene expression cassette. Blood. 1999;93:2208-2216.
7. Emery DW, Morrish F, Li Q, Stamatoyannopoulos G. Analysis of gamma-globin expression cassettes in retrovirus vectors. Hum Gene Ther. 1999;10:877-888.
8. Emery DW, Chen H, Li Q, Stamatoyannopoulos G. Development of a condensed locus control region cassette and testing in retrovirus vectors for A gamma-globin. Blood Cells Mol Dis. 1998;24:322-339.
9. Emery DW, Yannaki E, Tubb J, Nishino T, Li Q, Stamatoyannopoulos G. Development of virus vectors for gene therapy of beta chain hemoglobinopathies: flanking with a chromatin insulator reduces gamma-globin gene silencing in vivo. Blood. 2002;100:2012-2019.
10. Forrester WC, Novak U, Gelinas R, Groudine M. Molecular analysis of the human beta-globin locus activation region. Proc Natl Acad Sci U S A. 1989;86:5439-5443.
11. Li Q, Zhang M, Han H, Rohde A, Stamatoyannopoulos G. Evidence that DNase I hypersensitive site 5 of the human beta-globin locus control region functions as a chromosomal insulator in transgenic mice. Nucleic Acids Res. 2002;30:2484-2491.
12. Chung JH, Whiteley M, Felsenfeld G. A 5′ element of the chicken beta-globin domain serves as an insulator in human erythroid cells and protects against position effect in Drosophila. Cell. 1993;74:505-514.
13. Li Q, Stamatoyannopoulos JA. Position independence and proper developmental control of gamma-globin gene expression require both a 5′ locus control region and a downstream sequence element. Mol Cell Biol. 1994;14:6087-6096.
14. Duan ZJ, Fang X, Ronde A, Man H, Stamatoyannopoulos G, Li Q. Developmental specificity of recruitment of TBP to the TATA box of the human gamma-globin gene. Proc Natl Acad Sci U S A. 2002;99:5509-5514.
15. Thorpe SJ, Thein SL, Sampietro M, Craig JE, Mahon B, Huehns ER. Immunochemical estimation of haemoglobin types in red blood cells by FACS analysis. Br J Haematol. 1994;87:125-132.
16. Huang BL, Fan-Chiang IR, Wen SC, et al. Derepression of human embryonic zeta-globin promoter by a locus-control region sequence. Proc Natl Acad Sci U S A. 1998;95:14669-14674.
17. Li Q, Peterson KR, Fang X, Stamatoyannopoulos G. Locus control regions. Blood. 2002;100:3077-3086.
18. West AG, Gaszner M, Felsenfeld G. Insulators: many functions, many mechanisms. Genes Dev. 2002;16:271-288.
19. Hatton CS, Wilkie AO, Drysdale HC, et al. Alpha-thalassemia caused by a large (62 kb) deletion upstream of the human alpha globin gene cluster. Blood. 1990;76:221-227.
20. Jarman AP, Wood WG, Sharpe JA, Gourdon G, Ayyub H, Higgs DR. Characterization of the major regulatory element upstream of the human alpha-globin gene cluster. Mol Cell Biol. 1991;11:4679-4689.
21. Craddock CF, Vyas P, Sharpe JA, Ayyub H, Wood WG, Higgs DR. Contrasting effects of alpha and beta globin regulatory elements on chromatin structure may be related to their different chromosomal environments. EMBO J. 1995;14:1718-1726.
22. Barbour VM, Tufarelli C, Sharpe JA, et al. alpha-thalassemia resulting from a negative chromosomal position effect. Blood. 2000;96:800-807.
23. Emery DW, Yannaki E, Tubb J, Stamatoyannopoulos G. A chromatin insulator protects retrovirus vectors from chromosomal position effects. Proc Natl Acad Sci U S A. 2000;97:9150-9155.
24. Lung HY, Meeus IS, Weinberg RS, Atweh GF. In vivo silencing of the human gamma-globin gene in murine erythroid cells following retroviral transduction. Blood Cells Mol Dis. 2000;26:613-619.
25. Phillips RL, Ernst RE, Brunk B, et al. The genetic program of hematopoietic stem cells. Science. 2000;288:1635-1640.
26. Pannell D, Ellis J. Silencing of gene expression: implications for design of retrovirus vectors. Rev Med Virol. 2001;11:205-217.
27. Rivella S, Sadelain M. Genetic treatment of severe hemoglobinopathies: the combat against transgene variegation and transgene silencing. Semin Hematol. 1998;35:112-125.
28. Muller C, Leutz A. Chromatin remodeling in development and differentiation. Curr Opin Genet Dev. 2001;11:167-174.
29. Pannell D, Osborne CS, Yao S, et al. Retrovirus vector silencing is de novo methylase independent and marked by a repressive histone code. EMBO J. 2000;19:5884-5894.
30. Vassilopoulos G, Navas PA, Skarpidi E, et al. Correct function of the locus control region may require passage through a nonerythroid cellular environment. Blood. 1999;93:703-712.
31. May C, Rivella S, Callegari J, et al. Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin. Nature. 2000;406:82-86.
32. Pawliuk R, Westerman KA, Fabry ME, et al. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science. 2001;294:2368-2371.
33. Persons DA, Allay ER, Sawai N, et al. Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells. Blood. 2003;102:506-513.
34. Robertson G, Garrick D, Wu W, Kearns M, Martin D, Whitelaw E. Position-dependent variegation of globin transgene expression in mice. Proc Natl Acad Sci U S A. 1995;92:5371-5375.