Clinical Gene Therapy in Hematology: Past and Future

In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector

1996 • 4,854 citations

A Third-Generation Lentivirus Vector with a Conditional Packaging System

1998 • 3,175 citations

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

2000 • 2,632 citations

Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo

1997 • 1,977 citations

The Basic Science of Gene Therapy

1993 • 1,767 citations

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

1990 • 1,465 citations

T Lymphocyte-Directed Gene Therapy for ADA ⁻ SCID: Initial Trial Results After 4 Years

1995 • 1,464 citations

Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells.

1993 • 1,395 citations

Gene-marking to trace origin of relapse after autologous bone-marrow transplantation

1993 • 949 citations

Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA ⁻ Immunodeficient Patients

1995 • 846 citations

Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus

1994 • 798 citations

Identification of primitive human hematopoietic cells capable of repopulating NOD/SCID mouse bone marrow: Implications for gene therapy

1996 • 795 citations

Transduction of Human CD34 ⁺ Cells That Mediate Long-Term Engraftment of NOD/SCID Mice by HIV Vectors

1999 • 648 citations

Engraftment of gene–modified umbilical cord blood cells in neonates with adenosine deaminase deficiency

1995 • 622 citations

Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells

1996 • 565 citations

Genetic marking shows that Ph+ cells present in autologous transplants of chronic myelogenous leukemia (CML) contribute to relapse after autologous bone marrow in CML

1994 • 502 citations

Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector

1997 • 499 citations

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

1995 • 496 citations

Primitive Human Hematopoietic Cells Are Enriched in Cord Blood Compared With Adult Bone Marrow or Mobilized Peripheral Blood as Measured by the Quantitative In Vivo SCID-Repopulating Cell Assay

1997 • 488 citations

Gene marking to determine whether autologous marrow infusion restores long-term haemopoiesis in cancer patients

1993 • 434 citations

Direct demonstration that autologous bone marrow transplantation for solid tumors can return a multiplicity of tumorigenic cells

1994 • 418 citations

Stable transduction of quiescent CD34⁺CD38⁻human hematopoietic cells by HIV-1-based lentiviral vectors

1999 • 407 citations

Self-renewal of primitive human hematopoietic cells (long-term-culture-initiating cells) in vitro and their expansion in defined medium.

1996 • 393 citations

Transduction of nondividing cells using pseudotyped defective high-titer HIV type 1 particles

1996 • 384 citations

Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease

1997 • 347 citations

T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates

1998 • 334 citations

High-efficiency gene transfer into CD34+ cells with a human immunodeficiency virus type 1-based retroviral vector pseudotyped with vesicular stomatitis virus envelope glycoprotein G

1996 • 314 citations

HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G ₀ /G ₁ human hematopoietic stem cells

1998 • 306 citations

Expansion of human NOD/SCID-repopulating cells by stem cell factor, Flk2/Flt3 ligand, thrombopoietin, IL-6, and soluble IL-6 receptor

2000 • 282 citations

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

1990 • 273 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

Ex Vivo Expansion of Genetically Marked Rhesus Peripheral Blood Progenitor Cells Results in Diminished Long-Term Repopulating Ability

1998 • 257 citations

A Packaging Cell Line for Lentivirus Vectors

1999 • 257 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

Phase I trial of retroviral-mediated transfer of the human MDR1 gene as marrow chemoprotection in patients undergoing high-dose chemotherapy and autologous stem-cell transplantation.

1998 • 225 citations

In vivo selection of retrovirally transduced hematopoietic stem cells

1998 • 214 citations

Transduction of Human Progenitor Hematopoietic Stem Cells by Human Immunodeficiency Virus Type 1-Based Vectors Is Cell Cycle Dependent

1999 • 199 citations

Gene Transfer into Marrow Repopulating Cells: Comparison Between Amphotropic and Gibbon Ape Leukemia Virus Pseudotyped Retroviral Vectors in a Competitive Repopulation Assay in Baboons

1997 • 188 citations

Human Immunodeficiency Virus Type 1 Vectors Efficiently Transduce Human Hematopoietic Stem Cells

1998 • 176 citations

Retroviral Transfer of the Glucocerebrosidase Gene into CD34⁺Cells from Patients with Gaucher Disease:In VivoDetection of Transduced Cells without Myeloablation

1998 • 171 citations

Thrombopoietin, kit Ligand, and flk2/flt3 Ligand Together Induce Increased Numbers of Primitive Hematopoietic Progenitors From Human CD34+Thy-1+Lin− Cells With Preserved Ability to Engraft SCID-hu Bone

1998 • 170 citations

Efficient Retroviral-Mediated Gene Transfer to Human Cord Blood Stem Cells With In Vivo Repopulating Potential

1998 • 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

Retroviral-mediated gene transfer into CD34-enriched human peripheral blood stem cells.

1993 • 154 citations

Engraftment of MDR1 and NeoR Gene-Transduced Hematopoietic Cells After Breast Cancer Chemotherapy

1999 • 146 citations

Characterization of Membrane Components of the Erythrocyte Involved in Vesicular Stomatitis Virus Attachment and Fusion at Acidic pH

1987 • 140 citations

Engraftment of Hematopoietic Progenitor Cells Transduced with the Fanconi Anemia Group C Gene (FANCC)

1999 • 134 citations

Paclitaxel chemotherapy after autologous stem-cell transplantation and engraftment of hematopoietic cells transduced with a retrovirus containing the multidrug resistance complementary DNA (MDR1) in metastatic breast cancer patients.

1999 • 128 citations

Highly Efficient Transduction of the Green Fluorescent Protein Gene in Human Umbilical Cord Blood Stem Cells Capable of Cobblestone Formation in Long-Term Cultures and Multilineage Engraftment of Immunodeficient Mice

1998 • 115 citations

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

1999 • 100 citations

Distinct Requirements for Optimal Growth and In Vitro Expansion of Human CD34+CD38− Bone Marrow Long-Term Culture-Initiating Cells (LTC-IC), Extended LTC-IC, and Murine In Vivo Long-Term Reconstituting Stem Cells

1999 • 76 citations

Retroviral gene transfer to primitive normal and leukemic hematopoietic cells using clinically applicable procedures.

1992 • 73 citations

Retroviral Gene Transduction of Adult Peripheral Blood or Marrow-Derived CD34+ Cells for Six Hours Without Growth Factors or on Autologous Stroma Does Not Improve Marking Efficiency Assessed In Vivo

1997 • 70 citations

Human Growth Factor–Enhanced Regeneration of Transplantable Human Hematopoietic Stem Cells in Nonobese Diabetic/Severe Combined Immunodeficient Mice

1999 • 59 citations

Progress in Gene Therapy for Chronic Granulomatous Disease

1999 • 59 citations

The Use of Granulocyte Colony-Stimulating Factor During Retroviral Transduction on Fibronectin Fragment CH-296 Enhances Gene Transfer Into Hematopoietic Repopulating Cells in Dogs

1999 • 59 citations

Efficient Transduction of Human Lymphocytes and CD34+ Cells via Human Immunodeficiency Virus-Based Gene Transfer Vectors

1999 • 58 citations

A Murine Leukemia Virus (MuLV) Long Terminal Repeat Derived from Rhesus Macaques in the Context of a Lentivirus Vector and MuLV gag Sequence Results in High-Level Gene Expression in Human T Lymphocytes

2000 • 55 citations

Stable Integration of Human Immunodeficiency Virus-Based Retroviral Vectors into the Chromosomes of Nondividing Cells

1998 • 52 citations

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

2000 • 46 citations

Gene Marking

1996 • 42 citations

Feasibility of multidrug resistance (MDR-1) gene transfer in patients undergoing high-dose therapy and peripheral blood stem cell transplantation for lymphoma

1998 • 39 citations

Development of a Novel Selective Amplifier Gene for Controllable Expansion of Transduced Hematopoietic Cells

1997 • 37 citations

Chemotherapy immediately following autologous stem-cell transplantation in patients with advanced breast cancer.

1998 • 34 citations

Gene therapy for haematopoietic and lymphoid disorders.

1997 • 32 citations

Gene transfer approaches to the lysosomal storage disorders.

1999 • 30 citations

Viral vectors for gene therapy of hematopoietic cells

1997 • 28 citations

Gene transfer to hematopoietic cells — the clinical experience

1997 • 24 citations

Development of a modified selective amplifier gene for hematopoietic stem cell gene therapy

1999 • 20 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 (Proceedings of the National Academy of Sciences (December 24, 1996) 93 (15346-15351))

1997 • 6 citations