Treatment of genetic defects in hematopoietic cell function by gene transfer

Purification and Characterization of Mouse Hematopoietic Stem Cells

1988 • 2,856 citations

Improved retroviral vectors for gene transfer and expression.

1989 • 1,997 citations

Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection.

1990 • 1,465 citations

Redesign of Retrovirus Packaging Cell Lines To Avoid Recombination Leading to Helper Virus Production

1986 • 1,276 citations

Replacement Therapy for Inherited Enzyme Deficiency — Macrophage-Targeted Glucocerebrosidase for Gaucher's Disease

1991 • 1,243 citations

A safe packaging line for gene transfer: separating viral genes on two different plasmids

1988 • 1,118 citations

The molecular control of cell division, differentiation commitment and maturation in haemopoietic cells

1989 • 1,097 citations

Hemoglobin: Molecular, Genetic and Clinical Aspects.

1986 • 1,005 citations

Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges.

1988 • 908 citations

Characterization and partial purification of human marrow cells capable of initiating long-term hematopoiesis in vitro

1989 • 699 citations

Functional characterization of individual human hematopoietic stem cells cultured at limiting dilution on supportive marrow stromal layers.

1990 • 697 citations

Construction and use of a safe and efficient amphotropic packaging cell line

1988 • 637 citations

The new mouse genetics: Altering the genome by gene targeting

1989 • 608 citations

Clonal and systemic analysis of long-term hematopoiesis in the mouse.

1990 • 577 citations

Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells.

1986 • 533 citations

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

1984 • 527 citations

Progress Toward Human Gene Therapy

1989 • 484 citations

Identification and characterization of an inhibitor of haemopoietic stem cell proliferation

1990 • 470 citations

Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells.

1984 • 450 citations

HEMOPOIETIC CELL GROWTH FACTORS AND THEIR RECEPTORS

1989 • 432 citations

Evidence that the packaging signal of Moloney murine leukemia virus extends into the gag region

1987 • 426 citations

A Defective HSV-1 Vector Expresses Escherichia coli β-Galactosidase in Cultured Peripheral Neurons

1988 • 417 citations

Retrovirus Packaging Cells

1990 • 388 citations

Effect of internal viral sequences on the utility of retroviral vectors

1987 • 351 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

Resting and activated subsets of mouse multipotent hematopoietic stem cells.

1990 • 333 citations

Adenosine deaminase: characterization and expression of a gene with a remarkable promoter.

1985 • 329 citations

Combination of interleukins 3 and 6 preserves stem cell function in culture and enhances retrovirus-mediated gene transfer into hematopoietic stem cells.

1989 • 316 citations

Mechanisms that regulate the cell cycle status of very primitive hematopoietic cells in long-term human marrow cultures. II. Analysis of positive and negative regulators produced by stromal cells within the adherent layer

1991 • 292 citations

PREDICTION OF SEVERITY OF GAUCHER'S DISEASE BY IDENTIFICATION OF MUTATIONS AT DNA LEVEL

1989 • 284 citations

Gene Transfer by Retrovirus Vectors Occurs Only in Cells That Are Actively Replicating at the Time of Infection

1990 • 273 citations

MARROW TRANSPLANTATION FOR THALASSAEMIA FOLLOWING BUSULPHAN AND CYCLOPHOSPHAMIDE

1985 • 260 citations

Number and continuous proliferative pattern of transplanted primitive immunohematopoietic stem cells.

1988 • 220 citations

Cloning and Expression of Human Arylsulfatase A

1989 • 214 citations

Retrovirus-mediated transfer and expression of drug resistance genes in human haematopoietic progenitor cells

1986 • 213 citations

Molecular cloning and nucleotide sequence of human glucocerebrosidase cDNA.

1985 • 209 citations

Safety Issues Related to Retroviral-Mediated Gene Transfer in Humans

1991 • 207 citations

The Application of Bone Marrow Transplantation to the Treatment of Genetic Diseases

1986 • 207 citations

Improved gene expression upon transfer of the adenosine deaminase minigene outside the transcriptional unit of a retroviral vector.

1989 • 192 citations

DEVELOPMENTAL REGULATION OF HUMAN GLOBIN GENES

1985 • 190 citations

Expression of human adenosine deaminase in nonhuman primates after retrovirus-mediated gene transfer.

1987 • 187 citations

Development of a high-titer retrovirus producer cell line capable of gene transfer into rhesus monkey hematopoietic stem cells.

1990 • 186 citations

Clonal fluctuation within the haematopoietic system of mice reconstituted with retrovirus-infected stem cells.

1987 • 168 citations

Safety Considerations in Somatic Gene Therapy of Human Disease with Retrovirus Vectors

1990 • 150 citations

Isolation of cDNA clones encoding human acid sphingomyelinase: occurrence of alternatively processed transcripts.

1989 • 149 citations

Treatment of Late Infantile Metachromatic Leukodystrophy by Bone Marrow Transplantation

1990 • 149 citations

Adeno-associated virus vector for high-frequency integration, expression, and rescue of genes in mammalian cells.

1985 • 146 citations

Survival and retrovirus infection of murine hematopoietic stem cells in vitro: effects of 5-FU and method of infection.

1991 • 137 citations

Comparison of the Effects of Growth Factors on Retroviral Vector-Mediated Gene Transfer and the Proliferative Status of Human Hematopoietic Progenitor Cells

1990 • 133 citations

Long-term expression of human adenosine deaminase in mice transplanted with retrovirus-infected hematopoietic stem cells.

1989 • 133 citations

Gaucher disease: molecular heterogeneity and phenotype-genotype correlations.

1989 • 130 citations

An in Vivo Model of Somatic Cell Gene Therapy for Human Severe Combined Immunodeficiency

1991 • 129 citations

Nucleotide sequence of cDNA containing the complete coding sequence for human lysosomal glucocerebrosidase.

1986 • 129 citations

High-level beta-globin expression after retroviral transfer of locus activation region-containing human beta-globin gene derivatives into murine erythroleukemia cells.

1990 • 129 citations

Progress toward human gene therapy

1990 • 127 citations

Marrow Transplantation in Patients with Advanced Thalassemia

1987 • 126 citations

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

1988 • 119 citations

Expression of human adenosine deaminase from various strong promoters after gene transfer into human hematopoietic cell lines

1989 • 114 citations

Comparison of expression in hemopoietic cells by retroviral vectors carrying two genes

1988 • 111 citations

Progress toward human gene therapy

1990 • 109 citations

LONG-TERM FOLLOW-UP OF THE FIRST SUCCESSFUL BONE MARROW TRANSPLANTATION IN GAUCHER DISEASE

1988 • 108 citations

Stable expression of selectable genes introduced into human hematopoietic stem cells by electric field-mediated DNA transfer.

1986 • 108 citations

BONE-MARROW TRANSPLANTATION FOR INBORN ERROR OF PHAGOCYTIC CELLS ASSOCIATED WITH DEFECTIVE ADHERENCE, CHEMOTAXIS, AND OXIDATIVE RESPONSE DURING OPSONISED PARTICLE PHAGOCYTOSIS

1983 • 106 citations

Expression of human adenosine deaminase in mice reconstituted with retrovirus-transduced hematopoietic stem cells.

1990 • 105 citations

Purification of pluripotent hemopoietic stem cells: past and present.

1990 • 102 citations

Cell cycle dependence of synthesis of unintegrated viral DNA in mouse cells newly infected with murine leukemia virus

1981 • 101 citations

BENEFICIAL EFFECT OF PRE-TRANSPLANT SPLENECTOMY ON DISPLACEMENT BONE MARROW TRANSPLANTATION FOR GAUCHER'S SYNDROME

1987 • 98 citations

Canine model for gene therapy: inefficient gene expression in dogs reconstituted with autologous marrow infected with retroviral vectors

1988 • 96 citations

Expression of human adenosine deaminase in mice after transplantation of genetically-modified bone marrow

1990 • 93 citations

Effect of bone marrow transplantation on enzyme levels and clinical course in the neurologically affected twitcher mouse.

1988 • 93 citations

Correction of glucocerebrosidase deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with Gaucher disease.

1990 • 93 citations

Expression of Introduced Genetic Sequences in Hematopoietic Cells Following Retroviral-Mediated Gene Transfer

1990 • 92 citations

Retrovirus-mediated gene transfer of human adenosine deaminase: expression of functional enzyme in murine hematopoietic stem cells in vivo.

1987 • 89 citations

Retroviral transfer of genes into canine hemopoietic progenitor cells in culture: a model for human gene therapy.

1986 • 83 citations

Gene transfer into normal human hematopoietic cells using in vitro and in vivo assays

1991 • 82 citations

Treatment of Niemann-Pick disease type B by allogeneic bone marrow transplantation.

1987 • 76 citations

Retroviral vector-mediated high-efficiency expression of adenosine deaminase (ADA) in hematopoietic long-term cultures of ADA-deficient marrow cells.

1989 • 73 citations

The role of growth factors in self-renewal and differentiation of haemopoietic stem cells

1990 • 72 citations

In utero gene transfer and expression: a sheep transplantation model

1989 • 70 citations

Expression of human adenosine deaminase in mice transplanted with hemopoietic stem cells infected with amphotropic retroviruses.

1990 • 67 citations

Expression of the human beta-globin gene after retroviral transfer into murine erythroleukemia cells and human BFU-E cells.

1988 • 64 citations

Expression of genes transferred to haemopoietic stem cells by recombinant retroviruses.

1987 • 62 citations

Correction of CD18-Deficient Lymphocytes by Retrovirus-Mediated Gene Transfer

1990 • 60 citations

Transfer of genes into hematopoietic cells using recombinant DNA viruses.

1985 • 58 citations

Improved retroviral transfer of genes into canine hematopoietic progenitor cells kept in long-term marrow culture

1989 • 55 citations

A human purine nucleoside phosphorylase deficiency caused by a single base change.

1987 • 55 citations

Adenosine deaminase and purine nucleoside phosphorylase deficiencies: Evaluation of therapeutic interventions in eight patients

1987 • 54 citations

Fetal liver hematopoietic stem cells as a target for in utero retroviral gene transfer

1991 • 54 citations

Human adenosine deaminase expression in mice

1990 • 53 citations

Differential and synergistic effects of human granulocyte-macrophage colony-stimulating factor and human granulocyte colony-stimulating factor on hematopoiesis in human long-term marrow cultures

1991 • 50 citations

Murine leukemia inhibitory factor enhances retroviral-vector infection efficiency of hematopoietic progenitors

1990 • 50 citations

ADHESIVE PROPERTIES OF HAEMOPOIETIC STEM CELLS

1988 • 48 citations

Effects of Hemopoietic Growth Factors on Stem Cells in Vitro

1989 • 48 citations

Retroviral vector-mediated transfer of the bacterial neomycin resistance gene into fetal and adult sheep and human hematopoietic progenitors in vitro

1990 • 47 citations

Expression of a foreign gene in cats reconstituted with retroviral vector infected autologous bone marrow

1991 • 45 citations

High-efficiency gene transfer and expression in normal human hematopoietic cells with retrovirus vectors

1988 • 45 citations

Expression of human glucocerebrosidase in murine long-term bone marrow cultures after retroviral vector-mediated transfer

1990 • 43 citations

Differential and synergistic effects of human granulocyte-macrophage colony-stimulating factor and human granulocyte colony-stimulating factor on hematopoiesis in human long-term marrow cultures

1991 • 42 citations

Production of human glucocerebrosidase in mice after retroviral gene transfer into multipotential hematopoietic progenitor cells.

1989 • 41 citations

Gene Transfer of Adenosine Deaminase into Primitive Human Hematopoietic Progenitor Cells

1991 • 41 citations

Stable gene transfer and tissue-specific expression of a human globin gene using adenoviral vectors.

1986 • 38 citations

Gene therapy in perspective

1991 • 38 citations

Gene transfer into hematopoietic progenitor cells from normal and cyclic hematopoietic dogs using retroviral vectors

1988 • 37 citations

GENE THERAPY: CURRENT STATUS AND FUTURE DIRECTIONS

1990 • 37 citations

Transplantation of hematopoietic stem cells obtained by a combined dye method fractionation of murine bone marrow

1990 • 34 citations

Increased efficiency of retroviral-mediated gene transfer and expression in primate bone marrow progenitors after 5-fluorouracil- induced hematopoietic suppression and recovery

1991 • 29 citations

Enhancer sequences of a retroviral vector determine expression of a gene in multipotent hematopoietic progenitors and committed erythroid cells.

1987 • 28 citations

Bone marrow transplantation for metabolic disorders at Huddinge Hospital.

1990 • 27 citations

Expression of Human Glucocerebrosidase in Long-Term Reconstituted Mice Following Retroviral-Mediated Gene Transfer into Hematopoietic Stem Cells

1990 • 26 citations

Restriction of expression of an integrated recombinant retrovirus in primary but not immortalized murine hematopoietic stem cells

1988 • 25 citations

Long-term bone marrow cultures: their use in autologous marrow transplantation.

1989 • 24 citations

Expression of human alpha-globin and mouse/human hybrid beta-globin genes in murine hemopoietic stem cells transduced by recombinant retroviruses.

1990 • 23 citations

Targeted gene modification for gene therapy of stem cells

1990 • 21 citations

Molecular cloning and nucleotide sequence of human glucocerebrosidase cDNA

1986 • 19 citations

Clinical Considerations in the Design of Protocols for Somatic Gene Therapy

1991 • 19 citations

Gene transfer into hemopoietic stem cells using retroviral vectors

1989 • 19 citations

Displacement bone marrow transplantation and immunoprophylaxis for genetic diseases.

1988 • 19 citations

Increased efficiency of gene transfer with retroviral vectors in neonatal hematopoietic progenitor cells.

1990 • 18 citations

Developmental Regulation of Human Globin Genes

1985 • 17 citations

Gene transfer into normal human hematopoietic cells using in vitro and in vivo assays

1991 • 15 citations

Adeno-Associated Virus Vector for High-Frequency Integration, Expression, and Rescue of Genes in Mammalian Cells

1985 • 13 citations

Erythropoiesis in murine long-term marrow cultures following transfer of the erythropoietin cDNA into marrow stromal cells.

1990 • 13 citations

Retroviral transfer of genes into erythroid progenitors derived from human peripheral blood.

1990 • 11 citations

Gene transfer to primary normal and malignant human hemopoietic progenitors using recombinant retroviruses

1987 • 10 citations

Correction of enzyme deficiency by allogeneic bone marrow transplantation following total lymphoid irradiation in dogs with lysosomal storage disease (fucosidosis).

1986 • 10 citations

Improvement of Clinical Expression of Central Nervous System Manifestation in Lysosomal Storage Diseases Treated by Bone Marrow Transplantation

1988 • 9 citations

In utero gene transfer and expression: a sheep transplantation model

1989 • 9 citations

Improved retroviral transfer of genes into canine hematopoietic progenitor cells kept in long-term marrow culture

1989 • 8 citations

Hematopoietic Cell Growth Factors And Their Receptors

1989 • 8 citations

Transplantation of hematopoietic stem cells obtained by a combined dye method fractionation of murine bone marrow

1990 • 7 citations

Expression of human adenosine deaminase from various strong promoters after gene transfer into human hematopoietic cell lines

1989 • 6 citations

Reply to letter by Weissman et al.

1989 • 5 citations

Restriction of expression of an integrated recombinant retrovirus in primary but not immortalized murine hematopoietic stem cells

1988 • 5 citations

High-efficiency gene transfer and expression in normal human hematopoietic cells with retrovirus vectors

1988 • 4 citations

Fetal liver hematopoietic stem cells as a target for in utero retroviral gene transfer

1991 • 4 citations