Gene therapy for the hemoglobin disorders.

Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease

2000 • 2,632 citations

Sickle Cell Anemia, a Molecular Disease

1949 • 2,216 citations

Position-independent, high-level expression of the human β-globin gene in transgenic mice

1987 • 1,955 citations

Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning

2002 • 1,136 citations

Pathogenesis and Treatment of Sickle Cell Disease

1997 • 1,118 citations

A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells

1993 • 810 citations

Introduction of a selectable gene into primitive stem cells capable of long-term reconstitution of the hemopoietic system of W/W mice

1985 • 663 citations

The "beta-like-globin" gene domain in human erythroid cells.

1985 • 634 citations

Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin

2000 • 567 citations

Correction of Sickle Cell Disease in Transgenic Mouse Models by Gene Therapy

2001 • 563 citations

Introduction of new genetic material into pluripotent haematopoietic stem cells of the mouse

1984 • 527 citations

Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation

1995 • 496 citations

Incompletely reverse-transcribed human immunodeficiency virus type 1 genomes in quiescent cells can function as intermediates in the retroviral life cycle

1992 • 399 citations

A dominant control region from the human β-globin locus conferring integration site-independent gene expression

1989 • 346 citations

Lineage-specific expression of a human β-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells

1988 • 341 citations

Long-term engraftment of normal and post-5-fluorouracil murine marrow into normal nonmyeloablated mice [see comments]

1993 • 316 citations

The insulation of genes from external enhancers and silencing chromatin

2002 • 291 citations

Restriction of lentivirus in monkeys

2002 • 277 citations

Cellular inhibitors with Fv1-like activity restrict human and simian immunodeficiency virus tropism

2002 • 275 citations

Improved Gene Transfer Into Baboon Marrow Repopulating Cells Using Recombinant Human Fibronectin Fragment CH-296 in Combination With Interleukin-6, Stem Cell Factor, FLT-3 Ligand, and Megakaryocyte Growth and Development Factor

1998 • 262 citations

The level of mRNA encoding the amphotropic retrovirus receptor in mouse and human hematopoietic stem cells is low and correlates with the efficiency of retrovirus transduction.

1996 • 241 citations

Pathophysiology of thalassemia

2002 • 220 citations

A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human β-globin gene transfer

2003 • 218 citations

Comparison of Various Envelope Proteins for Their Ability to Pseudotype Lentiviral Vectors and Transduce Primitive Hematopoietic Cells from Human Blood

2002 • 203 citations

Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene.

1995 • 201 citations

The cHS4 Insulator Increases the Probability of Retroviral Expression at Random Chromosomal Integration Sites

2000 • 197 citations

Long-term survival of ex-thalassemic patients with persistent mixed chimerism after bone marrow transplantation

2000 • 189 citations

Correction of a mouse model of sickle cell disease: lentiviral/antisickling β-globin gene transduction of unmobilized, purified hematopoietic stem cells

2003 • 187 citations

Permanent and panerythroid correction of murine β thalassemia by multiple lentiviral integration in hematopoietic stem cells

2002 • 186 citations

Transduction of Human CD34+CD38- Bone Marrow and Cord Blood-Derived SCID-Repopulating Cells with Third-Generation Lentiviral Vectors

2000 • 182 citations

Abrogation of postentry restriction of HIV-1-based lentiviral vector transduction in simian cells

2003 • 173 citations

Mutagenesis of retroviral vectors transducing human beta-globin gene and beta-globin locus control region derivatives results in stable transmission of an active transcriptional structure.

1994 • 162 citations

Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus–based lentiviral vector system

2004 • 161 citations

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

2002 • 161 citations

Enhanced In Vivo Regenerative Potential of HOXB4-Transduced Hematopoietic Stem Cells With Regulation of Their Pool Size

1999 • 161 citations

Results of MDR-1 vector modification trial indicate that granulocyte/macrophage colony-forming unit cells do not contribute to posttransplant hematopoietic recovery following intensive systemic therapy

1996 • 160 citations

The degree of phenotypic correction of murine β-thalassemia intermedia following lentiviral-mediated transfer of a human γ-globin gene is influenced by chromosomal position effects and vector copy number

2003 • 159 citations

A correlation of erythrokinetics, ineffective erythropoiesis, and erythroid precursor apoptosis in Thai patients with thalassemia

2000 • 144 citations

Stem cell transplantation in the normal nonmyeloablated host: relationship between cell dose, schedule, and engraftment.

1997 • 138 citations

Successful treatment of murine β-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells

2003 • 126 citations

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

2000 • 121 citations

High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors

2001 • 119 citations

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

2001 • 119 citations

Expression of the human beta-globin gene following retroviral-mediated transfer into multipotential hematopoietic progenitors of mice.

1988 • 119 citations

Development of virus vectors for gene therapy of β chain hemoglobinopathies: flanking with a chromatin insulator reduces γ-globin gene silencing in vivo

2002 • 118 citations

Genetic treatment of severe hemoglobinopathies: the combat against transgene variegation and transgene silencing.

1998 • 112 citations

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

2000 • 111 citations

Marking and Gene Expression by a Lentivirus Vector in Transplanted Human and Nonhuman Primate CD34⁺Cells

2000 • 109 citations

Avoidance of stimulation improves engraftment of cultured and retrovirally transduced hematopoietic cells in primates

2001 • 105 citations

Highly efficient gene transfer into baboon marrow repopulating cells using GALV-pseudotype oncoretroviral vectors produced by human packaging cells

2002 • 103 citations

Efficient and Durable Gene Marking of Hematopoietic Progenitor Cells in Nonhuman Primates After Nonablative Conditioning

1999 • 100 citations

In vivo selection using a cell-growth switch

2000 • 97 citations

Engraftment of bone marrow cells into normal unprepared hosts: effects of 5-fluorouracil and cell cycle status

1995 • 96 citations

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

2001 • 89 citations

Gene therapy to protect haematopoietic cells from cytotoxic cancer drugs

2002 • 84 citations

Bone marrow transplantation in sickle cell anemia

2001 • 80 citations

Commentary on and reprint of Pauling L, Itano HA, Singer SJ, Wells IC, Sickle cell anemia, a molecular disease, in Science (1949) 110:543–548

2000 • 80 citations

Pharmacologically regulated in vivo selection in a large animal

2002 • 75 citations

Retroviral Marking and Transplantation of Rhesus Hematopoietic Cells by Nonmyeloablative Conditioning

1999 • 75 citations

Bone marrow transplantation (BMT) in 14 children with severe sickle cell disease (SCD): the French experience. GEGMO.

1993 • 73 citations

Introduction of a Xenogeneic Gene via Hematopoietic Stem Cells Leads to Specific Tolerance in a Rhesus Monkey Model

2000 • 72 citations

Lentivirus Vector-Mediated Hematopoietic Stem Cell Gene Transfer of Common Gamma-Chain Cytokine Receptor in Rhesus Macaques

2001 • 71 citations

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

1996 • 68 citations

Mixed chimerism in thalassemic patients after bone marrow transplantation.

1992 • 63 citations

Transcriptional Targeting of Retroviral Vectors to the Erythroblastic Progeny of Transduced Hematopoietic Stem Cells

1999 • 61 citations

Induction of Fetal Hemoglobin in Sickle Cell Disease

1999 • 61 citations

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

1997 • 61 citations

Lentivirus-mediated gene transfer into hematopoietic repopulating cells in baboons

2002 • 60 citations

Chimerism and cure: hematologic and pathologic correction of murine sickle cell disease

2003 • 58 citations

Sequences Flanking Hypersensitive Sites of the β-Globin Locus Control Region Are Required for Synergistic Enhancement

2001 • 49 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

2000 • 48 citations

Humoral immune response limits gene therapy in canine MPS I [letter]

1996 • 44 citations

In vivo selective expansion of gene-modified hematopoietic cells in a nonhuman primate model

2002 • 36 citations

Humoral immune response limits gene therapy in canine MPS I.

1996 • 32 citations

Persistent Low-Level Engraftment of Rhesus Peripheral Blood Progenitor Cells Transduced with the Fanconi Anemia C Gene after Conditioning with Low-Dose Irradiation

2001 • 32 citations

The effects of SCF/G-CSF prestimulation on radiation sensitivity and engraftment in nonmyeloablated murine hosts

2001 • 10 citations

In vivo expansion of gene‐modified hematopoietic cells by a novel selective amplifier gene utilizing the erythropoietin receptor as a molecular switch

2003 • 10 citations