Successful treatment of murine β-thalassemia intermedia by transfer of the human β-globin gene

Blood

Volumn 99, Issue 6, 2002, Pages 1902-1908

  May, Chad ^a,b,c   Rivella, Stefano ^a,b,c   Chadburn, Amy ^a,b,c   Sadelain, Michel ^a,b,c  

^a Immunology Program ^*  (United States)

^b MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

^c WEILL CORNELL MEDICAL COLLEGE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GLOBIN; LENTIVIRUS VECTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BETA THALASSEMIA; BLOOD TRANSFUSION; BONE MARROW CELL; BONE MARROW TRANSPLANTATION; CELL CULTURE; CHIMERA; CONTROLLED STUDY; DISEASE SEVERITY; ERYTHROPOIESIS; EXTRAMEDULLARY HEMATOPOIESIS; FEMALE; GENE EXPRESSION; GENE MUTATION; GENE TRANSFER; GENETIC CODE; GENETIC TRANSDUCTION; GENETIC TRANSFECTION; HEMOGLOBIN DETERMINATION; IRON STORAGE; MALE; NONHUMAN; PRIORITY JOURNAL; SPLEEN SIZE; TRANSGENE; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM;

EID: 0037085774     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.V99.6.1902     Document Type: Article

References (39)

1. The thalassemias
2. The thalassemias
3. Therapeutic approaches to hemoglobin switching in treatment of hemoglobinopathies
4. A series of cases of splenomegaly in children with anemia and peculiar bone changes
5. New trends in the treatment of beta-thalassemia
6. Bone marrow transplantation in thalassemia. The experience of Pesaro
7. Bone marrow transplantation for homozygous beta-thalassemia. The Memorial Sloan-Kettering Cancer Center experience
8. Gene therapy for the hemoglobin disorders: Past, present, and future
9. Genetic treatment of severe hemoglobinopathies: The combat against transgene variegation and transgene silencing
10. Toward gene therapy for disorders of hemoglobin synthesis
11. Issues in the manufacture and transplantation of genetically modified hematopoietic stem cells
12. Hematopoietic stem cell gene therapy: Towards clinically significant gene transfer efficiency
13. Preselection of retrovirally transduced bone marrow avoids subsequent stem cell gene silencing and age-dependent extinction of expression of human β-globin in engrafted mice
14. Lineage-specific expression of a human betaglobin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells
15. Expression of the human beta-globin gene following retroviral-mediated transfer into multipotential hematopoietic progenitors of mice
16. A majority of mice show long-term expression of a human beta-globin gene after retrovirus transfer into hematopoietic stem cells
17. Position-independent, high-level expression of the human beta-globin gene in transgenic mice
18. Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human betaglobin
19. A 3′ enhancer contributes to the stage-specific expression of the human beta-globin gene
20. Activation by locus control regions?
21. Looping, linking, and chromatin activity: New insights into beta-globin locus regulation
22. Mouse model for beta 0-thalassemia
23. Transgenic and gene knock-out mouse models of sickle cell anemia and the thalassemias
24. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
25. Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene
26. Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo
27. The human beta globin locus introduced by YAC transfer exhibits a specific and reproducible pattern of developmental regulation in transgenic mice
28. Compensatory splenic hemopoiesis in beta-thalassemic mice
29. Tissue iron deposition in untransfused beta-thalassemic mice
30. Phlebotomy to reduce iron overload in patients cured of thalassemia by bone marrow transplantation
31. Oral sodium phenylbutyrate therapy in homozygous beta thalassemia: A clinical trial
32. Viral vectors for gene therapy: The art of turning infectious agents into vehicles of therapeutics
33. Safety testing for replication-competent retrovirus associated with gibbon ape leukemia virus-pseudotyped retroviral vectors
34. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
35. Gamma-c gene transfer into SCID X1 patients' B-cell lines restores normal high-affinity interleukin-2 receptor expression and function
36. Functional requirements for phenotypic correction of murine beta-thalassemia: Implications for human gene therapy
37. In vivo selection of retrovirally transduced hematopoietic stem cells