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Results: 1 to 20 of 174

1.

Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin.

May C, Rivella S, Callegari J, Heller G, Gaensler KM, Luzzatto L, Sadelain M.

Nature. 2000 Jul 6;406(6791):82-6.

PMID:
10894546
[PubMed - indexed for MEDLINE]
2.
4.

Extended beta-globin locus control region elements promote consistent therapeutic expression of a gamma-globin lentiviral vector in murine beta-thalassemia.

Hanawa H, Hargrove PW, Kepes S, Srivastava DK, Nienhuis AW, Persons DA.

Blood. 2004 Oct 15;104(8):2281-90. Epub 2004 Jun 15.

PMID:
15198957
[PubMed - indexed for MEDLINE]
Free Article
5.

Permanent and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells.

Imren S, Payen E, Westerman KA, Pawliuk R, Fabry ME, Eaves CJ, Cavilla B, Wadsworth LD, Beuzard Y, Bouhassira EE, Russell R, London IM, Nagel RL, Leboulch P, Humphries RK.

Proc Natl Acad Sci U S A. 2002 Oct 29;99(22):14380-5. Epub 2002 Oct 21.

PMID:
12391330
[PubMed - indexed for MEDLINE]
Free PMC Article
6.

Current status of globin gene therapy for the treatment of beta-thalassaemia.

Lisowski L, Sadelain M.

Br J Haematol. 2008 May;141(3):335-45. doi: 10.1111/j.1365-2141.2008.07098.x. Review.

PMID:
18410569
[PubMed - indexed for MEDLINE]
7.

Successful correction of the human Cooley's anemia beta-thalassemia major phenotype using a lentiviral vector flanked by the chicken hypersensitive site 4 chromatin insulator.

Malik P, Arumugam PI, Yee JK, Puthenveetil G.

Ann N Y Acad Sci. 2005;1054:238-49. Review.

PMID:
16339671
[PubMed - indexed for MEDLINE]
8.

Progress toward the genetic treatment of the beta-thalassemias.

Sadelain M, Lisowski L, Samakoglu S, Rivella S, May C, Riviere I.

Ann N Y Acad Sci. 2005;1054:78-91. Review.

PMID:
16339654
[PubMed - indexed for MEDLINE]
9.

A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer.

Rivella S, May C, Chadburn A, Rivière I, Sadelain M.

Blood. 2003 Apr 15;101(8):2932-9. Epub 2002 Dec 12.

PMID:
12480689
[PubMed - indexed for MEDLINE]
Free Article
10.

Evaluation of optimal expression cassette in retrovirus vector for beta-thalassemia gene therapy.

Dong WJ, Li B, Liu DP, Zu ZX, Li J, Hao DL, Liu G, Guo ZC, Liang CC.

Mol Biotechnol. 2003 Jun;24(2):127-40.

PMID:
12746554
[PubMed - indexed for MEDLINE]
11.

A novel transgenic mouse model produced from lentiviral germline integration for the study of beta-thalassemia gene therapy.

Li W, Xie S, Guo X, Gong X, Wang S, Lin D, Zhang J, Ren Z, Huang S, Zeng F, Zeng Y.

Haematologica. 2008 Mar;93(3):356-62. doi: 10.3324/haematol.12010. Epub 2008 Feb 11.

PMID:
18268280
[PubMed - indexed for MEDLINE]
Free Article
12.

A promising genetic approach to the treatment of beta-thalassemia.

May C, Sadelain M.

Trends Cardiovasc Med. 2001 Oct;11(7):276-80. Review.

PMID:
11709281
[PubMed - indexed for MEDLINE]
13.

Amelioration of murine beta-thalassemia through drug selection of hematopoietic stem cells transduced with a lentiviral vector encoding both gamma-globin and the MGMT drug-resistance gene.

Zhao H, Pestina TI, Nasimuzzaman M, Mehta P, Hargrove PW, Persons DA.

Blood. 2009 Jun 4;113(23):5747-56. doi: 10.1182/blood-2008-10-186684. Epub 2009 Apr 13.

PMID:
19365082
[PubMed - indexed for MEDLINE]
Free PMC Article
14.

Gene Therapy for beta-thalassemia.

Malik P, Arumugam PI.

Hematology Am Soc Hematol Educ Program. 2005:45-50.

PMID:
16304358
[PubMed - indexed for MEDLINE]
Free Article
15.

Correction of a mouse model of sickle cell disease: lentiviral/antisickling beta-globin gene transduction of unmobilized, purified hematopoietic stem cells.

Levasseur DN, Ryan TM, Pawlik KM, Townes TM.

Blood. 2003 Dec 15;102(13):4312-9. Epub 2003 Aug 21.

PMID:
12933581
[PubMed - indexed for MEDLINE]
Free Article
16.

High-level beta-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells.

Imren S, Fabry ME, Westerman KA, Pawliuk R, Tang P, Rosten PM, Nagel RL, Leboulch P, Eaves CJ, Humphries RK.

J Clin Invest. 2004 Oct;114(7):953-62.

PMID:
15467834
[PubMed - indexed for MEDLINE]
Free PMC Article
17.

The beta-globin locus control region versus gene therapy vectors: a struggle for expression.

Ellis J, Pannell D.

Clin Genet. 2001 Jan;59(1):17-24. Review.

PMID:
11168020
[PubMed - indexed for MEDLINE]
18.

High-level transfer and long-term expression of the human beta-globin gene in a mouse transplant model.

Raftopoulos H, Ward M, Bank A.

Ann N Y Acad Sci. 1998 Jun 30;850:178-90.

PMID:
9668539
[PubMed - indexed for MEDLINE]
19.

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, Oh YH, Eaves CJ, Tuan DY, London IM.

EMBO J. 1994 Jul 1;13(13):3065-76.

PMID:
8039501
[PubMed - indexed for MEDLINE]
Free PMC Article
20.

Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells.

Persons DA, Allay ER, Sawai N, Hargrove PW, Brent TP, Hanawa H, Nienhuis AW, Sorrentino BP.

Blood. 2003 Jul 15;102(2):506-13. Epub 2003 Mar 27.

PMID:
12663444
[PubMed - indexed for MEDLINE]
Free Article

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