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Results: 1 to 20 of 438

1.

Gene replacement therapy for sickle cell disease and other blood disorders.

Townes TM.

Hematology Am Soc Hematol Educ Program. 2008:193-6. doi: 10.1182/asheducation-2008.1.193.

PMID:
19074080
[PubMed - indexed for MEDLINE]
Free Article
2.

Correction of sickle cell disease by homologous recombination in embryonic stem cells.

Wu LC, Sun CW, Ryan TM, Pawlik KM, Ren J, Townes TM.

Blood. 2006 Aug 15;108(4):1183-8. Epub 2006 Apr 25.

PMID:
16638928
[PubMed - indexed for MEDLINE]
Free PMC Article
3.

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
4.

Correction of sickle cell disease in transgenic mouse models by gene therapy.

Pawliuk R, Westerman KA, Fabry ME, Payen E, Tighe R, Bouhassira EE, Acharya SA, Ellis J, London IM, Eaves CJ, Humphries RK, Beuzard Y, Nagel RL, Leboulch P.

Science. 2001 Dec 14;294(5550):2368-71.

PMID:
11743206
[PubMed - indexed for MEDLINE]
Free Article
5.

Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin.

Hanna J, Wernig M, Markoulaki S, Sun CW, Meissner A, Cassady JP, Beard C, Brambrink T, Wu LC, Townes TM, Jaenisch R.

Science. 2007 Dec 21;318(5858):1920-3. Epub 2007 Dec 6.

PMID:
18063756
[PubMed - indexed for MEDLINE]
Free Article
6.

A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction.

Perumbeti A, Higashimoto T, Urbinati F, Franco R, Meiselman HJ, Witte D, Malik P.

Blood. 2009 Aug 6;114(6):1174-85. doi: 10.1182/blood-2009-01-201863. Epub 2009 May 27.

PMID:
19474450
[PubMed - indexed for MEDLINE]
Free PMC Article
7.

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
8.

Expression of an anti-sickling beta-globin in human erythroblasts derived from retrovirally transduced primitive normal and sickle cell disease hematopoietic cells.

Oh IH, Fabry ME, Humphries RK, Pawliuk R, Leboulch P, Hoffman R, Nagel RL, Eaves C.

Exp Hematol. 2004 May;32(5):461-9.

PMID:
15145214
[PubMed - indexed for MEDLINE]
9.

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]
10.

Hematopoietic stem cell gene transfer for the treatment of hemoglobin disorders.

Persons DA.

Hematology Am Soc Hematol Educ Program. 2009:690-7. doi: 10.1182/asheducation-2009.1.690. Review.

PMID:
20008255
[PubMed - indexed for MEDLINE]
Free Article
11.

Stem cell therapy for sickle cell disease: transplantation and gene therapy.

Walters MC.

Hematology Am Soc Hematol Educ Program. 2005:66-73.

PMID:
16304361
[PubMed - indexed for MEDLINE]
Free Article
12.

A phase I/II clinical trial of beta-globin gene therapy for beta-thalassemia.

Bank A, Dorazio R, Leboulch P.

Ann N Y Acad Sci. 2005;1054:308-16.

PMID:
16339679
[PubMed - indexed for MEDLINE]
13.

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
14.

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
15.

Engraftment of immune-deficient mice with primitive hematopoietic cells from beta-thalassemia and sickle cell anemia patients: implications for evaluating human gene therapy protocols.

Larochelle A, Vormoor J, Lapidot T, Sher G, Furukawa T, Li Q, Shultz LD, Olivieri NF, Stamatoyannopoulos G, Dick JE.

Hum Mol Genet. 1995 Feb;4(2):163-72.

PMID:
7757063
[PubMed - indexed for MEDLINE]
16.

Feasibility of using autologous transplantation to evaluate hematopoietic stem cell-based gene therapy strategies in transgenic mouse models of human disease.

Miller CL, Imren S, Antonchuk J, Kalberer C, Fabry ME, Nagel RL, Humphries RK, Eaves CJ.

Mol Ther. 2002 Sep;6(3):422-8.

PMID:
12231180
[PubMed - indexed for MEDLINE]
17.

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
18.

Gene therapy for the hemoglobin disorders.

Persons DA, Nienhuis AW.

Curr Hematol Rep. 2003 Jul;2(4):348-55. Review.

PMID:
12901333
[PubMed - indexed for MEDLINE]
19.

Gene transfer. A potential approach to gene therapy for sickle cell disease.

Bank A, Markowitz D, Lerner N.

Ann N Y Acad Sci. 1989;565:37-43. Review.

PMID:
2672970
[PubMed - indexed for MEDLINE]
20.

Polycistronic lentiviral vector for "hit and run" reprogramming of adult skin fibroblasts to induced pluripotent stem cells.

Chang CW, Lai YS, Pawlik KM, Liu K, Sun CW, Li C, Schoeb TR, Townes TM.

Stem Cells. 2009 May;27(5):1042-9. doi: 10.1002/stem.39.

PMID:
19415770
[PubMed - indexed for MEDLINE]
Free Article

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