-
1
-
-
0034624971
-
Gene therapy for the hemoglobin disorders: Past, present, and future
-
Persons DA, Nienhuis AW. Gene therapy for the hemoglobin disorders: past, present, and future. Proc Natl Acad Sci U S A. 2000;97:5022-5024.
-
(2000)
Proc Natl Acad Sci U S A
, vol.97
, pp. 5022-5024
-
-
Persons, D.A.1
Nienhuis, A.W.2
-
2
-
-
0036516375
-
Globin gene transfer for the treatment of severe hemoglobinopathies: A paradigm for stem cell-based gene therapy
-
Sadelain M. Globin gene transfer for the treatment of severe hemoglobinopathies: a paradigm for stem cell-based gene therapy. J Gene Med. 2002;4:113-121.
-
(2002)
J Gene Med
, vol.4
, pp. 113-121
-
-
Sadelain, M.1
-
3
-
-
0035164259
-
The beta-globin locus control region versus gene therapy vectors: A struggle for expression
-
Ellis J, Pannell D. The beta-globin locus control region versus gene therapy vectors: a struggle for expression. Clin Genet. 2001;59:17-24.
-
(2001)
Clin Genet
, vol.59
, pp. 17-24
-
-
Ellis, J.1
Pannell, D.2
-
4
-
-
0023663887
-
Position-independent, high-level expression of the human beta-globin gene in transgenic mice
-
Grosveld F, van Assendelft GB, Greaves DR, Kollias G. Position-independent, high-level expression of the human beta-globin gene in transgenic mice. Cell. 1987;51:975-985.
-
(1987)
Cell
, vol.51
, pp. 975-985
-
-
Grosveld, F.1
Van Assendelft, G.B.2
Greaves, D.R.3
Kollias, G.4
-
5
-
-
0027261194
-
Transgenic mice containing a 248-kb yeast artificial chromosome carrying the human beta-globin locus display proper developmental control of human globin genes
-
Peterson KR, Clegg CH, Huxley C, et al. Transgenic mice containing a 248-kb yeast artificial chromosome carrying the human beta-globin locus display proper developmental control of human globin genes. Proc Natl Acad Sci U S A. 1993;90:7593-7597.
-
(1993)
Proc Natl Acad Sci U S A
, vol.90
, pp. 7593-7597
-
-
Peterson, K.R.1
Clegg, C.H.2
Huxley, C.3
-
6
-
-
0027443269
-
Germ-line transmission and developmental regulation of a 150-kb yeast artificial chromosome containing the human beta-globin locus in transgenic mice
-
Gaensler KM, Kitamura M, Kan YW. Germ-line transmission and developmental regulation of a 150-kb yeast artificial chromosome containing the human beta-globin locus in transgenic mice. Proc Natl Acad Sci U S A. 1993;90:11381-11385.
-
(1993)
Proc Natl Acad Sci U S A
, vol.90
, pp. 11381-11385
-
-
Gaensler, K.M.1
Kitamura, M.2
Kan, Y.W.3
-
7
-
-
0024635998
-
A single erythroid-specific DNase I super-hypersensitive site activates high levels of human beta-globin gene expression in transgenic mice
-
Ryan TM, Behringer RR, Martin NC, Townes TM, Palmiter RD, Brinster RL. A single erythroid-specific DNase I super-hypersensitive site activates high levels of human beta-globin gene expression in transgenic mice. Genes Dev. 1989;3:314-323.
-
(1989)
Genes Dev
, vol.3
, pp. 314-323
-
-
Ryan, T.M.1
Behringer, R.R.2
Martin, N.C.3
Townes, T.M.4
Palmiter, R.D.5
Brinster, R.L.6
-
8
-
-
0029558513
-
Synergistic regulation of human beta-globin gene switching by locus control region elements HS3 and HS4
-
Bungert J, Dave U, Lim KC, et al. Synergistic regulation of human beta-globin gene switching by locus control region elements HS3 and HS4. Genes Dev. 1995;9:3083-3096.
-
(1995)
Genes Dev
, vol.9
, pp. 3083-3096
-
-
Bungert, J.1
Dave, U.2
Lim, K.C.3
-
11
-
-
0033609020
-
Conservation of sequence and structure flanking the mouse and human beta-globin loci: The beta-globin genes are embedded within an array of odorant receptor genes
-
Bulger M, van Doorninck JH, Saitoh N, et al. Conservation of sequence and structure flanking the mouse and human beta-globin loci: the beta-globin genes are embedded within an array of odorant receptor genes. Proc Natl Acad Sci U S A. 1999;96:5129-5134.
-
(1999)
Proc Natl Acad Sci U S A
, vol.96
, pp. 5129-5134
-
-
Bulger, M.1
Van Doorninck, J.H.2
Saitoh, N.3
-
12
-
-
0024506821
-
A dominant control region from the human beta-globin locus conferring integration site-independent gene expression
-
Talbot D, Collis P, Antoniou M, Vidal M, Grosveld F, Greaves DR. A dominant control region from the human beta-globin locus conferring integration site-independent gene expression. Nature. 1989;338:352-355.
-
(1989)
Nature
, vol.338
, pp. 352-355
-
-
Talbot, D.1
Collis, P.2
Antoniou, M.3
Vidal, M.4
Grosveld, F.5
Greaves, D.R.6
-
13
-
-
0030050402
-
Adominant chromatin-opening activity in 5′ hypersensitive site 3 of the human beta-globin locus control region
-
Ellis J, Tan-Un KC, Harper A, et al. Adominant chromatin-opening activity in 5′ hypersensitive site 3 of the human beta-globin locus control region. Embo J. 1996;15:562-568.
-
(1996)
Embo J
, vol.15
, pp. 562-568
-
-
Ellis, J.1
Tan-Un, K.C.2
Harper, A.3
-
14
-
-
0030819839
-
Evaluation of beta-globin gene therapy constructs in single copy transgenic mice
-
Ellis J, Pasceri P, Tan-Un KC, et al. Evaluation of beta-globin gene therapy constructs in single copy transgenic mice. Nucleic Acids Res. 1997;25:1296-1302.
-
(1997)
Nucleic Acids Res
, vol.25
, pp. 1296-1302
-
-
Ellis, J.1
Pasceri, P.2
Tan-Un, K.C.3
-
15
-
-
0343628721
-
Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human betaglobin
-
May C, Rivella S, Callegari J, et al. Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human betaglobin. Nature. 2000;406:82-86.
-
(2000)
Nature
, vol.406
, pp. 82-86
-
-
May, C.1
Rivella, S.2
Callegari, J.3
-
16
-
-
0037085774
-
Successful treatment of murine beta-thalassemia intermedia by transfer of the human beta-globin gene
-
May C, Rivella S, Chadburn A, Sadelain M. Successful treatment of murine beta-thalassemia intermedia by transfer of the human beta-globin gene. Blood. 2002;99:1902-1908.
-
(2002)
Blood
, vol.99
, pp. 1902-1908
-
-
May, C.1
Rivella, S.2
Chadburn, A.3
Sadelain, M.4
-
17
-
-
0035861452
-
Correction of sickle cell disease in transgenic mouse models by gene therapy
-
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.
-
(2001)
Science
, vol.294
, pp. 2368-2371
-
-
Pawliuk, R.1
Westerman, K.A.2
Fabry, M.E.3
-
18
-
-
0027473809
-
Synthetic human beta-globin 5′HS2 constructs function as locus control regions only in multicopy transgene concatamers
-
Ellis J, Talbot D, Dillon N, Grosveld F. Synthetic human beta-globin 5′HS2 constructs function as locus control regions only in multicopy transgene concatamers. Embo J. 1993;12:127-134.
-
(1993)
Embo J
, vol.12
, pp. 127-134
-
-
Ellis, J.1
Talbot, D.2
Dillon, N.3
Grosveld, F.4
-
19
-
-
0034657848
-
Locus control region activity by 5′HS3 requires a functional interaction with beta-globin gene regulatory elements: Expression of novel beta/gamma-globin hybrid transgenes
-
Rubin JE, Pasceri P, Wu X, Leboulch P, Ellis J. Locus control region activity by 5′HS3 requires a functional interaction with beta-globin gene regulatory elements: expression of novel beta/gamma-globin hybrid transgenes. Blood. 2000;95:3242-3249.
-
(2000)
Blood
, vol.95
, pp. 3242-3249
-
-
Rubin, J.E.1
Pasceri, P.2
Wu, X.3
Leboulch, P.4
Ellis, J.5
-
20
-
-
0029099173
-
The position of integration affects expression of the A gamma-globin-encoding gene linked to HS3 in transgenic mice
-
Navas PA, Josephson B, Furukawa T, Stamatoyannopoulos G, Li Q. The position of integration affects expression of the A gamma-globin-encoding gene linked to HS3 in transgenic mice. Gene. 1995;160:165-171.
-
(1995)
Gene
, vol.160
, pp. 165-171
-
-
Navas, P.A.1
Josephson, B.2
Furukawa, T.3
Stamatoyannopoulos, G.4
Li, Q.5
-
21
-
-
0028065129
-
Position independence and proper developmental control of gamma-globin gene expression require both a 5′ locus control region and a downstream sequence element
-
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.
-
(1994)
Mol Cell Biol
, vol.14
, pp. 6087-6096
-
-
Li, Q.1
Stamatoyannopoulos, J.A.2
-
22
-
-
0035874519
-
Functional requirements for phenotypic correction of murine beta-thalassemia: Implications for human gene therapy
-
Persons DA, Allay ER, Sabatino DE, Kelly P, Bodine DM, Nienhuis AW. Functional requirements for phenotypic correction of murine beta-thalassemia: implications for human gene therapy. Blood. 2001;97:3275-3282.
-
(2001)
Blood
, vol.97
, pp. 3275-3282
-
-
Persons, D.A.1
Allay, E.R.2
Sabatino, D.E.3
Kelly, P.4
Bodine, D.M.5
Nienhuis, A.W.6
-
23
-
-
0023955979
-
The human beta-globin gene contains multiple regulatory regions: Identification of one promoter and two downstream enhancers
-
Antoniou M, deBoer E, Habets G, Grosveld F. The human beta-globin gene contains multiple regulatory regions: identification of one promoter and two downstream enhancers. Embo J. 1988;7:377-384.
-
(1988)
Embo J
, vol.7
, pp. 377-384
-
-
Antoniou, M.1
DeBoer, E.2
Habets, G.3
Grosveld, F.4
-
24
-
-
0024293496
-
The human beta-globin promoter; nuclear protein factors and erythroid specific induction of transcription
-
deBoer E, Antoniou M, Mignotte V, Wall L, Grosveld F. The human beta-globin promoter; nuclear protein factors and erythroid specific induction of transcription. Embo J. 1988;7:4203-4212.
-
(1988)
Embo J
, vol.7
, pp. 4203-4212
-
-
DeBoer, E.1
Antoniou, M.2
Mignotte, V.3
Wall, L.4
Grosveld, F.5
-
25
-
-
0024114856
-
Nuclear scaffold attachment sites in the human globin gene complexes
-
Jarman AP, Higgs DR. Nuclear scaffold attachment sites in the human globin gene complexes. Embo J. 1988;7:3337-3344.
-
(1988)
Embo J
, vol.7
, pp. 3337-3344
-
-
Jarman, A.P.1
Higgs, D.R.2
-
27
-
-
0024080427
-
The human betaglobin gene 3′ enhancer contains multiple binding sites for an erythroid-specific protein
-
Wall L, deBoer E, Grosveld F. The human betaglobin gene 3′ enhancer contains multiple binding sites for an erythroid-specific protein. Genes Dev. 1988;2:1089-1100.
-
(1988)
Genes Dev
, vol.2
, pp. 1089-1100
-
-
Wall, L.1
DeBoer, E.2
Grosveld, F.3
-
28
-
-
0023442125
-
A 3′ enhancer contributes to the stage-specific expression of the human beta-globin gene
-
Trudel M, Costantini F. A 3′ enhancer contributes to the stage-specific expression of the human beta-globin gene. Genes Dev. 1987;1:954-961.
-
(1987)
Genes Dev
, vol.1
, pp. 954-961
-
-
Trudel, M.1
Costantini, F.2
-
29
-
-
0023547189
-
Two 3′ sequences direct adult erythroid-specific expression of human beta-globin genes in transgenic mice
-
Behringer RR, Hammer RE, Brinster RL, Palmiter RD, Townes TM. Two 3′ sequences direct adult erythroid-specific expression of human beta-globin genes in transgenic mice. Proc Natl Acad Sci U S A. 1987;84:7056-7060.
-
(1987)
Proc Natl Acad Sci U S A
, vol.84
, pp. 7056-7060
-
-
Behringer, R.R.1
Hammer, R.E.2
Brinster, R.L.3
Palmiter, R.D.4
Townes, T.M.5
-
30
-
-
0035525735
-
High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with selfinactivating lentiviral vectors
-
Moreau-Gaudry F, Xia P, Jiang G, et al. High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with selfinactivating lentiviral vectors. Blood. 2001;98:2664-2672.
-
(2001)
Blood
, vol.98
, pp. 2664-2672
-
-
Moreau-Gaudry, F.1
Xia, P.2
Jiang, G.3
-
31
-
-
0028303758
-
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
-
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.
-
(1994)
Embo J
, vol.13
, pp. 3065-3076
-
-
Leboulch, P.1
Huang, G.M.2
Humphries, R.K.3
-
32
-
-
0029084785
-
Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene
-
Sadelain M, Wang CH, Antoniou M, Grosveld F, Mulligan RC. Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene. Proc Natl Acad Sci U S A. 1995;92:6728-6732.
-
(1995)
Proc Natl Acad Sci U S A
, vol.92
, pp. 6728-6732
-
-
Sadelain, M.1
Wang, C.H.2
Antoniou, M.3
Grosveld, F.4
Mulligan, R.C.5
-
33
-
-
0028803688
-
Nuclear factor binding sites in human beta globin IVS2
-
Jackson CE, O'Neill D, Bank A. Nuclear factor binding sites in human beta globin IVS2. J Biol Chem. 1995;270:28448-28456.
-
(1995)
J Biol Chem
, vol.270
, pp. 28448-28456
-
-
Jackson, C.E.1
O'Neill, D.2
Bank, A.3
-
34
-
-
0023684064
-
A general method of in vitro preparation and specific mutagenesis of DNA fragments: Study of protein and DNA interactions
-
Higuchi R, Krummel B, Saiki RK. A general method of in vitro preparation and specific mutagenesis of DNA fragments: study of protein and DNA interactions. Nucleic Acids Res. 1988;16:7351-7367.
-
(1988)
Nucleic Acids Res
, vol.16
, pp. 7351-7367
-
-
Higuchi, R.1
Krummel, B.2
Saiki, R.K.3
-
35
-
-
0022549802
-
Regulated expression of human A gamma-, beta-, and hybrid gamma beta-globin genes in transgenic mice: Manipulation of the developmental expression patterns
-
Kollias G, Wrighton N, Hurst J, Grosveld F. Regulated expression of human A gamma-, beta-, and hybrid gamma beta-globin genes in transgenic mice: manipulation of the developmental expression patterns. Cell. 1986;46:89-94.
-
(1986)
Cell
, vol.46
, pp. 89-94
-
-
Kollias, G.1
Wrighton, N.2
Hurst, J.3
Grosveld, F.4
-
36
-
-
0029863093
-
A palindromic regulatory site within vertebrate GATA-1 promoters requires both zinc fingers of the GATA-1 DNA-binding domain for high-affinity interaction
-
Trainor CD, Omichinski JG, Vandergon TL, Gronenborn AM, Clore GM, Felsenfeld G. A palindromic regulatory site within vertebrate GATA-1 promoters requires both zinc fingers of the GATA-1 DNA-binding domain for high-affinity interaction. Mol Cell Biol. 1996;16:2238-2247.
-
(1996)
Mol Cell Biol
, vol.16
, pp. 2238-2247
-
-
Trainor, C.D.1
Omichinski, J.G.2
Vandergon, T.L.3
Gronenborn, A.M.4
Clore, G.M.5
Felsenfeld, G.6
-
37
-
-
0028877438
-
The POU homeodomain transcription factor Oct-1 is essential for activity of the gonadotropin-releasing hormone neuronspecific enhancer
-
Clark ME, Mellon PL. The POU homeodomain transcription factor Oct-1 is essential for activity of the gonadotropin-releasing hormone neuronspecific enhancer. Mol Cell Biol. 1995;15:6169-6177.
-
(1995)
Mol Cell Biol
, vol.15
, pp. 6169-6177
-
-
Clark, M.E.1
Mellon, P.L.2
-
38
-
-
0032529521
-
Oct-1 is involved in the transcriptional repression of the von Willebrand factor gene promoter
-
Schwachtgen JL, Remacle JE, Janel N, et al. Oct-1 is involved in the transcriptional repression of the von Willebrand factor gene promoter. Blood. 1998;92:1247-1258.
-
(1998)
Blood
, vol.92
, pp. 1247-1258
-
-
Schwachtgen, J.L.1
Remacle, J.E.2
Janel, N.3
-
39
-
-
0025806272
-
Complex pattern of immunoglobulin mu gene expression in normal and transgenic mice: Nonoverlapping regulatory sequences govern distinct tissue specificities
-
Jenuwein T, Grosschedl R. Complex pattern of immunoglobulin mu gene expression in normal and transgenic mice: nonoverlapping regulatory sequences govern distinct tissue specificities. Genes Dev. 1991;5:932-943.
-
(1991)
Genes Dev
, vol.5
, pp. 932-943
-
-
Jenuwein, T.1
Grosschedl, R.2
-
40
-
-
0027522759
-
The minimal requirements for activity in transgenic mice of hypersensitive site 3 of the beta globin locus control region
-
Philipsen S, Pruzina S, Grosveld F. The minimal requirements for activity in transgenic mice of hypersensitive site 3 of the beta globin locus control region. Embo J. 1993;12:1077-1085.
-
(1993)
Embo J
, vol.12
, pp. 1077-1085
-
-
Philipsen, S.1
Pruzina, S.2
Grosveld, F.3
-
41
-
-
0034623079
-
GATA zinc finger interactions modulate DNA binding and transactivation
-
Trainor CD, Ghirlando R, Simpson MA. GATA zinc finger interactions modulate DNA binding and transactivation. J Biol Chem. 2000;275:28157-28166.
-
(2000)
J Biol Chem
, vol.275
, pp. 28157-28166
-
-
Trainor, C.D.1
Ghirlando, R.2
Simpson, M.A.3
-
42
-
-
0032511224
-
Perturbation of nucleosome structure by the erythroid transcription factor GATA-1
-
Boyes J, Omichinski J, Clark D, Pikaart M, Felsenfeld G. Perturbation of nucleosome structure by the erythroid transcription factor GATA-1. J Mol Biol. 1998;279:529-544.
-
(1998)
J Mol Biol
, vol.279
, pp. 529-544
-
-
Boyes, J.1
Omichinski, J.2
Clark, D.3
Pikaart, M.4
Felsenfeld, G.5
-
43
-
-
0034662013
-
Human gamma-globin gene promoter element regulates human beta-globin gene developmental specificity
-
Ryan TM, Sun CW, Ren J, Townes TM. Human gamma-globin gene promoter element regulates human beta-globin gene developmental specificity. Nucleic Acids Res. 2000;28:2736-2740.
-
(2000)
Nucleic Acids Res
, vol.28
, pp. 2736-2740
-
-
Ryan, T.M.1
Sun, C.W.2
Ren, J.3
Townes, T.M.4
-
44
-
-
0032709770
-
Nuclear matrix attachment regions antagonize methylation-dependent repression of long-range enhancer-promoter interactions
-
Forrester WC, Fernandez LA, Grosschedl R. Nuclear matrix attachment regions antagonize methylation-dependent repression of long-range enhancer-promoter interactions. Genes Dev. 1999;13:3003-3014.
-
(1999)
Genes Dev
, vol.13
, pp. 3003-3014
-
-
Forrester, W.C.1
Fernandez, L.A.2
Grosschedl, R.3
-
45
-
-
0034750803
-
Matrix attachment region-dependent function of the immunoglobulin mu enhancer involves histone acetylation at a distance without changes in enhancer occupancy
-
Fernandez LA, Winkler M, Grosschedl R. Matrix attachment region-dependent function of the immunoglobulin mu enhancer involves histone acetylation at a distance without changes in enhancer occupancy. Mol Cell Biol. 2001;21:196-208.
-
(2001)
Mol Cell Biol
, vol.21
, pp. 196-208
-
-
Fernandez, L.A.1
Winkler, M.2
Grosschedl, R.3
-
46
-
-
0031030535
-
Extension of chromatin accessibility by nuclear matrix attachment regions
-
Jenuwein T, Forrester WC, Fernandez-Herrero LA, Laible G, Dull M, Grosschedl R. Extension of chromatin accessibility by nuclear matrix attachment regions. Nature. 1997;385:269-272.
-
(1997)
Nature
, vol.385
, pp. 269-272
-
-
Jenuwein, T.1
Forrester, W.C.2
Fernandez-Herrero, L.A.3
Laible, G.4
Dull, M.5
Grosschedl, R.6
-
47
-
-
0034161939
-
The MAR-binding protein SATB1 orchestrates temporal and spatial expression of multiple genes during T-cell development
-
Alvarez JD, Yasui DH, Niida H, Joh T, Loh DY, Kohwi-Shigematsu T. The MAR-binding protein SATB1 orchestrates temporal and spatial expression of multiple genes during T-cell development. Genes Dev. 2000;14:521-535.
-
(2000)
Genes Dev
, vol.14
, pp. 521-535
-
-
Alvarez, J.D.1
Yasui, D.H.2
Niida, H.3
Joh, T.4
Loh, D.Y.5
Kohwi-Shigematsu, T.6
-
49
-
-
0031564633
-
Identification and characterization of genomic nucleosome-positioning sequences
-
Widlund HR, Cao H, Simonsson S, et al. Identification and characterization of genomic nucleosome-positioning sequences. J Mol Biol. 1997;267:807-817.
-
(1997)
J Mol Biol
, vol.267
, pp. 807-817
-
-
Widlund, H.R.1
Cao, H.2
Simonsson, S.3
-
50
-
-
0030964249
-
Nucleosome mobility and the maintenance of nucleosome positioning
-
Pazin MJ, Bhargava P, Geiduschek EP, Kadonaga JT. Nucleosome mobility and the maintenance of nucleosome positioning. Science. 1997;276:809-812.
-
(1997)
Science
, vol.276
, pp. 809-812
-
-
Pazin, M.J.1
Bhargava, P.2
Geiduschek, E.P.3
Kadonaga, J.T.4
-
51
-
-
0028233376
-
Nucleosome positioning on chicken and human globin gene promoters in vitro: Novel mapping techniques
-
Yenidunya A, Davey C, Clark D, Felsenfeld G, Allan J. Nucleosome positioning on chicken and human globin gene promoters in vitro: novel mapping techniques. J Mol Biol. 1994;237:401-414.
-
(1994)
J Mol Biol
, vol.237
, pp. 401-414
-
-
Yenidunya, A.1
Davey, C.2
Clark, D.3
Felsenfeld, G.4
Allan, J.5
-
52
-
-
0037057668
-
SATB1 targets chromatin remodelling to regulate genes over long distances
-
Yasui D, Miyano M, Cai S, Varga-Weisz P, Kohwi-Shigematsu T. SATB1 targets chromatin remodelling to regulate genes over long distances. Nature. 2002;419:641-645.
-
(2002)
Nature
, vol.419
, pp. 641-645
-
-
Yasui, D.1
Miyano, M.2
Cai, S.3
Varga-Weisz, P.4
Kohwi-Shigematsu, T.5
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