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Items: 1 to 20 of 48

1.

Globin lentiviral vector insertions can perturb the expression of endogenous genes in beta-thalassemic hematopoietic cells.

Hargrove PW, Kepes S, Hanawa H, Obenauer JC, Pei D, Cheng C, Gray JT, Neale G, Persons DA.

Mol Ther. 2008 Mar;16(3):525-33. doi: 10.1038/sj.mt.6300394. Epub 2008 Jan 15.

2.
3.

Improved human beta-globin expression from self-inactivating lentiviral vectors carrying the chicken hypersensitive site-4 (cHS4) insulator element.

Arumugam PI, Scholes J, Perelman N, Xia P, Yee JK, Malik P.

Mol Ther. 2007 Oct;15(10):1863-71. Epub 2007 Jul 10.

4.

Preservation of spleen and brain function in children with sickle cell anemia treated with hydroxyurea.

Hankins JS, Helton KJ, McCarville MB, Li CS, Wang WC, Ware RE.

Pediatr Blood Cancer. 2008 Feb;50(2):293-7.

PMID:
17554794
5.

Extended core sequences from the cHS4 insulator are necessary for protecting retroviral vectors from silencing position effects.

Aker M, Tubb J, Groth AC, Bukovsky AA, Bell AC, Felsenfeld G, Kiem HP, Stamatoyannopoulos G, Emery DW.

Hum Gene Ther. 2007 Apr;18(4):333-43.

PMID:
17411365
6.

Pathophysiology and therapy for haemoglobinopathies. Part I: sickle cell disease.

Madigan C, Malik P.

Expert Rev Mol Med. 2006 Apr 28;8(9):1-23. Review.

PMID:
16690007
7.

A nucleolin-binding 3' untranslated region element stabilizes beta-globin mRNA in vivo.

Jiang Y, Xu XS, Russell JE.

Mol Cell Biol. 2006 Mar;26(6):2419-29.

8.

A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference.

Samakoglu S, Lisowski L, Budak-Alpdogan T, Usachenko Y, Acuto S, Di Marzo R, Maggio A, Zhu P, Tisdale JF, Rivière I, Sadelain M.

Nat Biotechnol. 2006 Jan;24(1):89-94. Epub 2005 Dec 25.

PMID:
16378095
10.

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

Walters MC.

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

PMID:
16304361
11.

The response of autologous T cells to a human melanoma is dominated by mutated neoantigens.

Lennerz V, Fatho M, Gentilini C, Frye RA, Lifke A, Ferel D, Wölfel C, Huber C, Wölfel T.

Proc Natl Acad Sci U S A. 2005 Nov 1;102(44):16013-8. Epub 2005 Oct 24.

12.

Long-term hydroxyurea therapy for infants with sickle cell anemia: the HUSOFT extension study.

Hankins JS, Ware RE, Rogers ZR, Wynn LW, Lane PA, Scott JP, Wang WC.

Blood. 2005 Oct 1;106(7):2269-75.

13.

Pathology of Berkeley sickle cell mice: similarities and differences with human sickle cell disease.

Manci EA, Hillery CA, Bodian CA, Zhang ZG, Lutty GA, Coller BS.

Blood. 2006 Feb 15;107(4):1651-8. Epub 2005 Sep 15.

14.

The Corfu deltabeta thalassemia deletion disrupts gamma-globin gene silencing and reveals post-transcriptional regulation of HbF expression.

Chakalova L, Osborne CS, Dai YF, Goyenechea B, Metaxotou-Mavromati A, Kattamis A, Kattamis C, Fraser P.

Blood. 2005 Mar 1;105(5):2154-60. Epub 2004 Nov 9.

15.

Gene therapy for the hemoglobin disorders.

Persons DA, Tisdale JF.

Semin Hematol. 2004 Oct;41(4):279-86. Review.

PMID:
15508113
16.

Sickle cell anemia a molecular disease.

PAULING L, ITANO HA, et al.

Science. 1949 Nov 25;110(2865):543-8. No abstract available.

PMID:
15395398
17.

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.

18.

Pois(s)on--it's a question of dose...

Fehse B, Kustikova OS, Bubenheim M, Baum C.

Gene Ther. 2004 Jun;11(11):879-81. Review. No abstract available.

PMID:
15057265
19.
20.

Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia.

Iannone R, Casella JF, Fuchs EJ, Chen AR, Jones RJ, Woolfrey A, Amylon M, Sullivan KM, Storb RF, Walters MC.

Biol Blood Marrow Transplant. 2003 Aug;9(8):519-28.

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