Functional requirements for phenotypic correction of murine β-thalassemia: implications for human gene therapy

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Mouse model of human beta zero thalassemia: targeted deletion of the mouse beta maj- and beta min-globin genes in embryonic stem cells.

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Limiting numbers of G156A O6-methylguanine–DNA methyltransferase-transduced marrow progenitors repopulate nonmyeloablated mice after drug selection

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Protection and in Vivo Selection of Hematopoietic Stem Cells Using Temozolomide, O6-Benzylguanine, and an Alkyltransferase-Expressing Retroviral Vector

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Prolonged High-Level Detection of Retrovirally Marked Hematopoietic Cells in Nonhuman Primates after Transduction of CD34+ Progenitors Using Clinically Feasible Methods

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A human beta-globin gene fused to the human beta-globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus-transduced hematopoietic stem cells

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GRAFT-VERSUS-HOST DISEASE AFTER BONE MARROW TRANSPLANTATION FOR THALASSEMIA

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A 46 base pair enhancer sequence within the locus activating region is required for induced expression of the gamma-globin gene during erythroid differentiation

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Enhanced engraftment of hematopoietic progenitor cells in mice treated with granulocyte colony-stimulating factor before low-dose irradiation: implications for gene therapy.

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Mixed chimerism in thalassemic patients after bone marrow transplantation.

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Long-Term Transfer and Expression of the Human β-Globin Gene in a Mouse Transplant Model

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Production of genetically stable high-titer retroviral vectors that carry a human gamma-globin gene under the control of the alpha-globin locus control region

1996 • 56 citations

Long-term expression of γ-globin mRNA in mouse erythrocytes from retrovirus vectors containing the human γ-globin gene fused to the ankyrin-1 promoter

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A Minimal Ankyrin Promoter Linked to a Human γ-Globin Gene Demonstrates Erythroid Specific Copy Number Dependent Expression with Minimal Position or Enhancer Dependence in Transgenic Mice

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Expansion of genetically modified primary human hemopoietic cells using chemical inducers of dimerization

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Multiple regions of p45 NF-E2 are required for -globin gene expression in erythroid cells

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Substitution of the Human β-Spectrin Promoter for the Human ^Aγ-Globin Promoter Prevents Silencing of a Linked Human β-Globin Gene in Transgenic Mice

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Gene therapy for the hemoglobin disorders: Past, present, and future

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Analysis of beta-globin mutations shows stable mixed chimerism in patients with thalassemia after bone marrow transplantation

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Regulation of human fetal and adult globin genes in mouse erythroleukemia cells

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Analysis of gamma-Globin Expression Cassettes in Retrovirus Vectors

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Hematology of a Murine β‐Thalassemia: A Longitudinal Study^a

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Hepatitis C virus infection in thalassemia patients undergoing allogeneic bone marrow transplantation.

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CURE OF MURINE THALASSEMIA BY BONE MARROW TRANSPLANTATION WITHOUT ERADICATION OF ENDOGENOUS STEM CELLS

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15 The Hematopoietic System as a Target for Gene Therapy

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DNA fragments of the Mus musculus beta globin haplotypes Hbbs and Hbbd.

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Hematopoietic repopulation of adult mice with beta-thalassemia

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Correction of murine beta-thalassemia by partial bone marrow chimerism: selective advantage of normal erythropoiesis.

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Selective advantage of normal erythrocyte production after bone marrow transplantation of alpha-thalassemic mice.

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Hematopoietic repopulation of adult mice with beta-thalassemia

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Thalassaemia clinical and patho-physiological considerations

1967 • 7 citations