Format
Sort by
Items per page

Send to

Choose Destination

Links from PubMed

Items: 1 to 20 of 103

1.

Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients.

Roselli EA, Mezzadra R, Frittoli MC, Maruggi G, Biral E, Mavilio F, Mastropietro F, Amato A, Tonon G, Refaldi C, Cappellini MD, Andreani M, Lucarelli G, Roncarolo MG, Marktel S, Ferrari G.

EMBO Mol Med. 2010 Aug;2(8):315-28. doi: 10.1002/emmm.201000083.

2.

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.

3.

Bone marrow as a source of hematopoietic stem cells for human gene therapy of β-thalassemia.

Frittoli MC, Biral E, Cappelli B, Zambelli M, Roncarolo MG, Ferrari G, Ciceri F, Marktel S.

Hum Gene Ther. 2011 Apr;22(4):507-13. doi: 10.1089/hum.2010.045. Epub 2011 Mar 4.

PMID:
20979441
4.

Lentiviral Transfer of γ-Globin with Fusion Gene NUP98-HOXA10HD Expands Hematopoietic Stem Cells and Ameliorates Murine β-Thalassemia.

Zhao HF, Abraham A, Kim YS, Wang YD, Pestina T, Zhan J, Humphries K, Nienhuis AW, Persons DA.

Mol Ther. 2017 Mar 1;25(3):593-605. doi: 10.1016/j.ymthe.2017.01.019. Epub 2017 Feb 9.

PMID:
28190779
6.

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.

7.

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
8.

In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of beta-thalassemia.

Miccio A, Cesari R, Lotti F, Rossi C, Sanvito F, Ponzoni M, Routledge SJ, Chow CM, Antoniou MN, Ferrari G.

Proc Natl Acad Sci U S A. 2008 Jul 29;105(30):10547-52. doi: 10.1073/pnas.0711666105. Epub 2008 Jul 23.

9.

The new self-inactivating lentiviral vector for thalassemia gene therapy combining two HPFH activating elements corrects human thalassemic hematopoietic stem cells.

Papanikolaou E, Georgomanoli M, Stamateris E, Panetsos F, Karagiorga M, Tsaftaridis P, Graphakos S, Anagnou NP.

Hum Gene Ther. 2012 Jan;23(1):15-31. doi: 10.1089/hum.2011.048. Epub 2011 Dec 5.

10.

Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients.

Breda L, Casu C, Gardenghi S, Bianchi N, Cartegni L, Narla M, Yazdanbakhsh K, Musso M, Manwani D, Little J, Gardner LB, Kleinert DA, Prus E, Fibach E, Grady RW, Giardina PJ, Gambari R, Rivella S.

PLoS One. 2012;7(3):e32345. doi: 10.1371/journal.pone.0032345. Epub 2012 Mar 27.

11.

Genetic therapy for beta-thalassemia: from the bench to the bedside.

Arumugam P, Malik P.

Hematology Am Soc Hematol Educ Program. 2010;2010:445-50. doi: 10.1182/asheducation-2010.1.445.

PMID:
21239833
12.

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
13.

Gene Therapy for beta-thalassemia.

Malik P, Arumugam PI.

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

PMID:
16304358
14.

Gaining the hard yard: pre-clinical evaluation of lentiviral-mediated gene therapy for the treatment of beta-thalassemia.

Milsom MD, Williams DA.

EMBO Mol Med. 2010 Aug;2(8):291-3. doi: 10.1002/emmm.201000086.

16.

Parallel assessment of globin lentiviral transfer in induced pluripotent stem cells and adult hematopoietic stem cells derived from the same transplanted β-thalassemia patient.

Tubsuwan A, Abed S, Deichmann A, Kardel MD, Bartholomä C, Cheung A, Negre O, Kadri Z, Fucharoen S, von Kalle C, Payen E, Chrétien S, Schmidt M, Eaves CJ, Leboulch P, Maouche-Chrétien L.

Stem Cells. 2013 Sep;31(9):1785-94. doi: 10.1002/stem.1436.

17.

Successful correction of the human beta-thalassemia major phenotype using a lentiviral vector.

Puthenveetil G, Scholes J, Carbonell D, Qureshi N, Xia P, Zeng L, Li S, Yu Y, Hiti AL, Yee JK, Malik P.

Blood. 2004 Dec 1;104(12):3445-53. Epub 2004 Aug 3.

18.

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.

19.

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.

20.

Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease.

Negre O, Bartholomae C, Beuzard Y, Cavazzana M, Christiansen L, Courne C, Deichmann A, Denaro M, de Dreuzy E, Finer M, Fronza R, Gillet-Legrand B, Joubert C, Kutner R, Leboulch P, Maouche L, Paulard A, Pierciey FJ, Rothe M, Ryu B, Schmidt M, von Kalle C, Payen E, Veres G.

Curr Gene Ther. 2015;15(1):64-81.

Supplemental Content

Support Center