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Items: 1 to 20 of 302

1.

Progress toward safe and effective gene therapy for beta-thalassemia and sickle cell disease.

Lebensburger J, Persons DA.

Curr Opin Drug Discov Devel. 2008 Mar;11(2):225-32. Review.

PMID:
18283610
2.

Development of gene therapy for hemoglobin disorders.

Nienhuis AW, Hanawa H, Sawai N, Sorrentino BP, Persons DA.

Ann N Y Acad Sci. 2003 May;996:101-11.

PMID:
12799288
3.

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
4.

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
5.

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
6.

Genetic correction of sickle cell anemia and beta-thalassemia: progress and new perspective.

Perumbeti A, Malik P.

ScientificWorldJournal. 2010 Apr 13;10:644-54. doi: 10.1100/tsw.2010.67. Review.

7.

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
9.
10.

Gene therapy for the hemoglobin disorders.

Persons DA, Nienhuis AW.

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

PMID:
12901333
11.

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.

12.

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.

13.

The challenge of obtaining therapeutic levels of genetically modified hematopoietic stem cells in beta-thalassemia patients.

Persons DA.

Ann N Y Acad Sci. 2010 Aug;1202:69-74. doi: 10.1111/j.1749-6632.2010.05581.x. Review.

PMID:
20712775
14.
15.

Update on gene therapy for hemoglobin disorders.

Persons DA.

Curr Opin Mol Ther. 2003 Oct;5(5):508-16. Review.

PMID:
14601520
16.

Future prospects for treatment of hemoglobinopathies.

Stamatoyannopoulos JA.

West J Med. 1992 Dec;157(6):631-6. Review.

17.

Hemoglobin gene therapy for β-thalassemia.

Bank A.

Hematol Oncol Clin North Am. 2010 Dec;24(6):1187-201. doi: 10.1016/j.hoc.2010.08.002. Review.

PMID:
21075288
18.

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.

19.

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.

Blood. 2001 May 15;97(10):3275-82.

20.

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.

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