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Results: 1 to 20 of 26

1.

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
2.

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]
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
[PubMed - indexed for MEDLINE]
Free Article
4.

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]
5.

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
[PubMed - indexed for MEDLINE]
6.

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
[PubMed - indexed for MEDLINE]
7.

Recent advances in globin gene transfer for the treatment of beta-thalassemia and sickle cell anemia.

Sadelain M.

Curr Opin Hematol. 2006 May;13(3):142-8. Review.

PMID:
16567956
[PubMed - indexed for MEDLINE]
8.

Stem cell engineering for the treatment of severe hemoglobinopathies.

Sadelain M, Boulad F, Lisowki L, Moi P, Riviere I.

Curr Mol Med. 2008 Nov;8(7):690-7. Review.

PMID:
18991654
[PubMed - indexed for MEDLINE]
9.

[Improvements of lentiviral vector and its application in gene therapy of hematological diseases].

Zhu HH, Xu KL.

Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2003 Apr;11(2):208-12. Review. Chinese.

PMID:
12744751
[PubMed - indexed for MEDLINE]
10.

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
[PubMed - indexed for MEDLINE]
11.

Therapeutic globin gene delivery using lentiviral vectors.

Rivella S, Sadelain M.

Curr Opin Mol Ther. 2002 Oct;4(5):505-14. Review.

PMID:
12435053
[PubMed - indexed for MEDLINE]
12.

Vector design for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells.

Schambach A, Baum C.

DNA Repair (Amst). 2007 Aug 1;6(8):1187-96. Epub 2007 May 7. Review.

PMID:
17482894
[PubMed - indexed for MEDLINE]
Free PMC Article
13.

Towards more successful gene therapy clinical trials for β-thalassemia.

Drakopoulou E, Papanikolaou E, Georgomanoli M, Anagnou NP.

Curr Mol Med. 2013 Sep;13(8):1314-30. Review.

PMID:
23865429
[PubMed - indexed for MEDLINE]
14.

Live and let die: in vivo selection of gene-modified hematopoietic stem cells via MGMT-mediated chemoprotection.

Milsom MD, Williams DA.

DNA Repair (Amst). 2007 Aug 1;6(8):1210-21. Epub 2007 May 7. Review.

PMID:
17482893
[PubMed - indexed for MEDLINE]
Free PMC Article
15.

Advances in stem cell transplantation and gene therapy in the β-hemoglobinopathies.

Payen E, Leboulch P.

Hematology Am Soc Hematol Educ Program. 2012;2012:276-83. doi: 10.1182/asheducation-2012.1.276. Review.

PMID:
23233592
[PubMed - indexed for MEDLINE]
Free Article
16.

Development of gene therapy for hematopoietic stem cells using lentiviral vectors.

Woods NB, Ooka A, Karlsson S.

Leukemia. 2002 Apr;16(4):563-9. Review.

PMID:
11960333
[PubMed - indexed for MEDLINE]
Free Article
17.

Alternative options for DNA-based experimental therapy of β-thalassemia.

Gambari R.

Expert Opin Biol Ther. 2012 Apr;12(4):443-62. doi: 10.1517/14712598.2012.665047. Review.

PMID:
22413823
[PubMed - indexed for MEDLINE]
18.

Genetic strategies for the treatment of sickle cell anaemia.

Mansilla-Soto J, Rivière I, Sadelain M.

Br J Haematol. 2011 Sep;154(6):715-27. doi: 10.1111/j.1365-2141.2011.08773.x. Epub 2011 Jun 28. Review.

PMID:
21707580
[PubMed - indexed for MEDLINE]
19.

Major challenges for gene therapy of thalassemia and sickle cell disease.

Papanikolaou E, Anagnou NP.

Curr Gene Ther. 2010 Oct;10(5):404-12. Review.

PMID:
20712578
[PubMed - indexed for MEDLINE]
20.

Development of gene therapy for thalassemia.

Nienhuis AW, Persons DA.

Cold Spring Harb Perspect Med. 2012 Nov 1;2(11). pii: a011833. doi: 10.1101/cshperspect.a011833. Review.

PMID:
23125203
[PubMed - indexed for MEDLINE]
Free PMC Article
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