Gene Therapy for Adenosine Deaminase Deficiency

The retinoblastoma protein and cell cycle control

1995 • 4,873 citations

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

1996 • 4,854 citations

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

1995 • 1,464 citations

Interleukin-2 receptor γ chain mutation results in X-linked severe combined immunodeficiency in humans

1993 • 1,317 citations

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

1995 • 846 citations

Versatile retroviral vectors for potential use in gene therapy.

1994 • 690 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

Prospects for Human Gene Therapy

1984 • 548 citations

Fibronectin and VLA-4 in haematopoietic stem cell–microenvironment interactions

1991 • 522 citations

A functional comparison of CD34 + CD38- cells in cord blood and bone marrow

1995 • 456 citations

Cytostatic Gene Therapy for Vascular Proliferative Disorders with a Constitutively Active form of the Retinoblastoma Gene Product

1995 • 455 citations

Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo.

1994 • 415 citations

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

1999 • 407 citations

Treatment of thromboangiitis obliterans (Buerger's disease) by intramuscular gene transfer of vascular endothelial growth factor: Preliminary clinical results

1998 • 390 citations

HIV nuclear import is governed by the phosphotyrosine-mediated binding of matrix to the core domain of integrase

1995 • 360 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

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

A Phase I Study of an Adeno-Associated Virus-CFTR Gene Vector in Adult CF Patients with Mild Lung Disease. Johns Hopkins Children's Center, Baltimore, Maryland

1996 • 265 citations

Combined immunodeficiency disease associated with adenosine deaminase deficiency

1975 • 255 citations

Severe Combined Immunodeficiency and Adenosine Deaminase Deficiency

1975 • 251 citations

Immune response in human melanoma after transfer of an allogeneic class I major histocompatibility complex gene with DNA–liposome complexes

1996 • 237 citations

Spontaneous in vivo reversion to normal of an inherited mutation in a patient with adenosine deaminase deficiency

1996 • 229 citations

Bone marrow extracellular matrix molecules improve gene transfer into human hematopoietic cells via retroviral vectors.

1994 • 228 citations

Multiple modifications in cis elements of the long terminal repeat of retroviral vectors lead to increased expression and decreased DNA methylation in embryonic carcinoma cells

1995 • 211 citations

Fibronectin improves transduction of reconstituting hematopoietic stem cells by retroviral vectors: evidence of direct viral binding to chymotryptic carboxy-terminal fragments

1996 • 207 citations

Transduction of pluripotent human hematopoietic stem cells demonstrated by clonal analysis after engraftment in immune-deficient mice.

1996 • 186 citations

Bone marrow gene transfer in three patients with adenosine deaminase deficiency.

1996 • 185 citations

Analysis of optimal conditions for retroviral-mediated transduction of primitive human hematopoietic cells

1995 • 182 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

Adhesion to Fibronectin Maintains Regenerative Capacity During Ex Vivo Culture and Transduction of Human Hematopoietic Stem and Progenitor Cells

1998 • 168 citations

FLT3 Ligand Preserves the Ability of Human CD34+ Progenitors to Sustain Long-Term Hematopoiesis in Immune-Deficient Mice After Ex Vivo Retroviral-Mediated Transduction

1997 • 161 citations

Thymic lymphoproliferative disease after successful correction of CD40 ligand deficiency by gene transfer in mice

1998 • 148 citations

Consistent, persistent expression from modified retroviral vectors in murine hematopoietic stem cells

1998 • 143 citations

Restoration of lymphocyte function in Janus Kinase 3-deficient mice by retroviral-mediated gene transfer

1998 • 143 citations

Isolation of a recombinant murine leukemia virus utilizing a new primer tRNA

1986 • 129 citations

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

1991 • 129 citations

High-Efficiency Gene Transfer into Normal and Adenosine Deaminase-Deficient T Lymphocytes Is Mediated by Transduction on Recombinant Fibronectin Fragments

1998 • 124 citations

Adenosine deaminase deficiency: clinical expression, molecular basis, and therapy.

1998 • 123 citations

Increased probability of expression from modified retroviral vectors in embryonal stem cells and embryonal carcinoma cells

1997 • 123 citations

Reduction in levels of the cyclin-dependent kinase inhibitor p27 ^kip-1 coupled with transforming growth factor β neutralization induces cell-cycle entry and increases retroviral transduction of primitive human hematopoietic cells

1998 • 119 citations

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

1988 • 111 citations

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

1987 • 89 citations

Ex Vivo Gene Therapy of Familial Hypercholesterolemia. Howard Hughes Medical Institute, University of Michigan

1992 • 83 citations

Negative control region at the 5' end of murine leukemia virus long terminal repeats.

1989 • 49 citations

Retroviral Mediated Gene Transfer of the Fanconi Anemia Complementation Group C Gene to Hematopoietic Progenitors of Group C Patients. National Institutes of Health, Bethesda, Maryland

1997 • 39 citations

Evidence for engraftment of donor-type multipotent CD34+ cells in a patient with selective T-lymphocyte reconstitution after bone marrow transplantation for B-SCID

1994 • 38 citations

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

1998 • 37 citations

Gene therapy for congenital lymphoid immunodeficiency diseases.

1998 • 18 citations

Fibronectin improves transduction of reconstituting hematopoietic stem cells by retroviral vectors: evidence of direct viral binding to chymotryptic carboxy-terminal fragments

1996 • 10 citations

A quantitative ranking system for relating genotype to phenotype in adenosine deaminase (ADA) deficiency, based on the expressed activity of 29 mutant alleles in E. coli

1998 • 3 citations