Gene therapy for chronic granulomatous disease

NADPH Oxidase: An Update

1999 • 2,134 citations

Mouse model of X–linked chronic granulomatous disease, an inherited defect in phagocyte superoxide production

1995 • 920 citations

Hematology of Infancy and Childhood.

1975 • 918 citations

A Controlled Trial of Interferon Gamma to Prevent Infection in Chronic Granulomatous Disease

1991 • 785 citations

Versatile retroviral vectors for potential use in gene therapy.

1994 • 690 citations

The Molecular Basis of Blood Diseases.

1987 • 648 citations

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

1996 • 565 citations

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

1984 • 527 citations

The p47phox mouse knock-out model of chronic granulomatous disease.

1995 • 487 citations

Mutations in the X-linked and autosomal recessive forms of chronic granulomatous disease

1996 • 424 citations

Absence of Respiratory Burst in X-linked Chronic Granulomatous Disease Mice Leads to Abnormalities in Both Host Defense and Inflammatory Response to Aspergillus fumigatus

1997 • 370 citations

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

1997 • 347 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

Recent Advances in Chronic Granulomatous Disease

1983 • 236 citations

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

1994 • 228 citations

Treatment of genetic defects in hematopoietic cell function by gene transfer

1991 • 225 citations

In vivo selection of retrovirally transduced hematopoietic stem cells

1998 • 214 citations

Gene targeting of X chromosome-linked chronic granulomatous disease locus in a human myeloid leukemia cell line and rescue by expression of recombinant gp91phox.

1993 • 213 citations

Embryonic stem cell virus, a recombinant murine retrovirus with expression in embryonic stem cells.

1990 • 203 citations

Enhanced Host Defense After Gene Transfer in the Murine p47phox-Deficient Model of Chronic Granulomatous Disease

1997 • 133 citations

The NADPH oxidase and chronic granulomatous disease

1996 • 130 citations

Chronic Granulomatous Disease Presenting in a 69-Year-Old Man

1991 • 127 citations

Retroviral-Mediated Gene Transfer of gp91phox Into Bone Marrow Cells Rescues Defect in Host Defense Against Aspergillus fumigatus in Murine X-Linked Chronic Granulomatous Disease

1997 • 124 citations

Expression of retroviral vectors containing the human multidrug resistance 1 cDNA in hematopoietic cells of transplanted mice

1995 • 110 citations

Retroviral-Mediated Gene Transfer of gp91phox Into Bone Marrow Cells Rescues Defect in Host Defense Against Aspergillus fumigatus in Murine X-Linked Chronic Granulomatous Disease

1997 • 103 citations

Prognosis of chronic granulomatous disease.

1990 • 93 citations

Continuous therapy with sulfamethoxazole-trimethoprim in patients with chronic granulomatous disease

1983 • 88 citations

Use of an X-linked human neutrophil marker to estimate timing of lyonization and size of the dividing stem cell pool.

1985 • 86 citations

Peripheral blood progenitors as a target for genetic correction of p47phox-deficient chronic granulomatous disease.

1993 • 85 citations

X-linked Inheritance in Females with Chronic Granulomatous Disease

1980 • 83 citations

CD34+ peripheral blood progenitors as a target for genetic correction of the two flavocytochrome b558 defective forms of chronic granulomatous disease

1994 • 83 citations

Long-Term Correction of Phagocyte NADPH Oxidase Activity by Retroviral-Mediated Gene Transfer in Murine X-Linked Chronic Granulomatous Disease

1999 • 82 citations

GENE TRANSFER TO HEMATOPOIETIC STEM CELLS: Implications for Gene Therapy of Human Disease

1996 • 72 citations

In vitro molecular reconstitution of the respiratory burst in B lymphoblasts from p47-phox-deficient chronic granulomatous disease.

1993 • 70 citations

A study of 25 patients with chronic granulomatous disease: A new classification by correlating respiratory burst, cytochrome b, and flavoprotein

1986 • 63 citations

Clinical differences in chronic granulomatous disease in patients with cytochrome b-negative or cytochrome b-positive neutrophils

1985 • 62 citations

Chronic granulomatous disease with partial deficiency of cytochrome b558 and incomplete respiratory burst: variants of the X-linked, cytochrome b558-negative form of the disease

1992 • 60 citations

Marrow transplantation in chronic granulomatous disease: An update, with 6-year follow-up

1988 • 60 citations

High-level reconstitution of respiratory burst activity in a human X- linked chronic granulomatous disease (X-CGD) cell line and correction of murine X-CGD bone marrow cells by retroviral-mediated gene transfer of human gp91phox

1996 • 59 citations

A new X-linked variant of chronic granulomatous disease characterized by the existence of a normal clone of respiratory burst-competent phagocytic cells

1995 • 57 citations

X-linked chronic granulomatous disease: correction of NADPH oxidase defect by retrovirus-mediated expression of gp91-phox

1993 • 50 citations

Restitution of superoxide generation in autosomal cytochrome-negative chronic granulomatous disease (A22(0) CGD)-derived B lymphocyte cell lines by transfection with p22phax cDNA.

1993 • 50 citations

Genetic Correction of p67phox Deficient Chronic Granulomatous Disease Using Peripheral Blood Progenitor Cells as a Target for Retrovirus Mediated Gene Transfer

1997 • 46 citations

Molecular basis of the autosomal recessive forms of chronic granulomatous disease.

1992 • 46 citations

Molecular analysis in three cases of X91- variant chronic granulomatous disease

1995 • 44 citations

Successful bone marrow transplantation in a child with X-linked chronic granulomatous disease.

1996 • 42 citations

Retrovirus-mediated reconstitution of respiratory burst activity in X- linked chronic granulomatous disease cells

1994 • 41 citations

Recurrent severe infections in a girl with apparently variable expression of mosaicism for chronic granulomatous disease

1985 • 40 citations

Chronic granulomatous disease 100% corrected by displacement bone marrow transplantation from a volunteer unrelated donor

1992 • 36 citations

Bone marrow transplantation in chronic granulomatous disease

1996 • 31 citations

Transgenic mice expressing the tyr22 variant of murine DHFR: protection of transgenic marrow transplant recipients from lethal doses of methotrexate.

1997 • 30 citations

Efficient retroviral transduction of human bone marrow progenitor and long-term culture-initiating cells: partial reconstitution of cells from patients with X-linked chronic granulomatous disease by gp91-phox expression

1996 • 25 citations

Hematopoietic stem cell gene therapy: a current overview.

1999 • 16 citations

GENE THERAPY FOR CONGENITAL IMMUNODEFICIENCY DISEASES

1996 • 11 citations

Partial reconstitution of the respiratory burst oxidase in lymphoblastoid B cell lines lacking p67-phox after transfection with an expression vector containing wild-type and mutant p67 -phox cDNAs: Deletions of the carboxy and amino terminal residues of p67-phox are not required for activity.

1996 • 10 citations