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Article
What is the status of gene therapy for primary immunodeficiency?
The efforts to find satisfactory treatments for seriously ill patients with primary immunodeficiency have resulted in the development of important new therapeutic procedures with benefits reaching far beyond t...
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Article
Use of a herpes thymidine kinase/neomycin phosphotransferase chimeric gene for metabolic suicide gene transfer
Metabolic suicide gene transfer is widely applied for gene therapy of cancer, and retroviral vectors expressing the herpes simplex virus thymidine kinase (HSV-tk) gene are commonly used in clinical trials. Most o...
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Article
Gene therapy of primary immunodeficiencies
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Article
Peripheral expansion of pre-existing mature T cells is an important means of CD4+ T-cell regeneration HIV-infected adults
The CD4+ T-cell pool in HIV-infected patients is in a constant state of flux as CD4+ T cells are infected and destroyed by HIV and new cells take their place. To study T-cell survival, we adoptively transferred p...
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Article
T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates
Adenosine deaminase-deficient severe combined immunodeficiency was the first disease investigated for gene therapy because of a postulated production or survival advantage for genecorrected T lymphocytes, whic...
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Chapter and Conference Paper
Gene Therapy for Cancer
Cancer is a complex acquired disease that affects millions of individuals each year. Underlying the pathogenesis of cancer are a variety of molecular genetic abnormalities which can be inherited or environment...
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Article
Therapy of malignant brain tumors by intratumoral implantation of retroviral vector-producing cells
Intratumoral implantation of murine cells modified to produce retroviral vectors containing the herpes simplex virus-thymidine kinase (HSV-TK) gene induces regression of experimental brain tumors in rodents after...
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Article
SPLICE SITE MUTATIONS IN THE WISKOTT ALDRICH SYNDROME. • 59
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Article
Engraftment of gene–modified umbilical cord blood cells in neonates with adenosine deaminase deficiency
Haematopoietic stem cells in umbilical cord blood are an attractive target for gene therapy of inborn errors of metabolism. Three neonates with severe combined immunodeficiency were treated by retroviral–media...
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Chapter
Gene Therapy for Adenosine Deaminase Deficiency and Malignant Solid Tumors
Gene therapy may be considered the ultimate treatment for genetic diseases (Anderson, 1984). The possibility of reversing genetic defects by replacing the defective gene instead of merely treating symptoms is ...
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Article
Development of Gene Therapy for Immunodeficiency: Adenosine Deaminase Deficiency
ABSTRACT: Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency. Clinical cure has been observed in several ADA-severe combined immunodeficiency patients after bone marrow transpl...
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Chapter and Conference Paper
Gene Therapy for Immunodeficiency and Cancer
Gene transfer technology has enormous potential for the treatment of both inherited “genetic” disorders as well as diseases not ordinarily thought of as “genetic.” Since initial studies would be restricted to ex ...
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Article
Retrovirus-mediated gene transfer into CD4+ and CD8+ human T cell subsets derived from tumor-infiltrating lymphocytes and peripheral blood mononuclear cells
Studies were undertaken to test the susceptibility of individual T cell subpopulations to retroviral-mediated gene transduction. Gene transfer into human tumor-infiltrating lymphocytes (TIL) or peripheral bloo...
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Chapter
Retroviral-Mediated Gene Transfer into Hemopoietic Cells
Retroviral vectors have provided a means for the introduction of functioning exogenous genes into the hematopoietic system of whole animals. Although these vectors are quite efficient in the mouse model, when ...
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Article
ESTABLISHMENT AND CHARACTERIZATION OF ADENOSINE DEAMINASE (ADA)-DEFICIENT T CELL LINES
Congenital deficiency of the purine metabolic enzyme ADA causes severe combined immunodeficiency (SCID). The profound T lymphopenia characteristic of this disease has limited direct investigation of the cell m...
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Article
FAVORABLE RESPONSE TO IVIgG IN A WISCOTT-ALDRICH SYNDROME INFANT WITH SEVERE THROMBOCYTOPENIA REFRACTORY TO SPLENECTOMY
Other than bone marrow transplantation, splenectomy is the only effective, long term therapy for the severe thrombocytopenia of Wiscott-Aldrich Syndrome (WAS). Occasional WAS patients demonstrate continued sev...
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Article
WISKOTT-ALDRICH SYNDROME (WAS) CARRIER DETECTION BY X-CHROMOSOME INACTIVATION ANALYSIS
WAS is an X-linked disorder characterized by immune deficiency, thrombocytopenia and eczema. Previous studies of G-6-PD isozyme expression in rare females doubly heterozygous for WAS and G-6-PD found one of tw...
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Article
EVALUATION OF RETROVIRAL MEDIATED GENE TRANSFER BY IN SITU HYBRIDIZATION
Genetically engineered retroviral vectors can be used to transfer genes into eucaryotic cells. The application of these vectors for human gene therapy is being evaluated in vitro and in animal models. The time co...
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Article
920 EFFECTS OF STEROID HORMONES ON IMMUNOGLOBULIN PRODUCTION IN ADULT AMD CORD BLOOD LYMPHOCYTES
The effect of steroid compounds on the in vitro production of immunoglobulins was evaluated in cultures of peripheral blood mononuclear cells (PBMC). In a reverse hemolytic plaque assay the addition of 10-5 to 10
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Article
IMMUNODEFICIENCY IN LYMPHOHISTIOCYTOSIS: DEFECTIVE CELLULAR IMMUNITY IN ASSOCIATION WITH HYPERLIPIDEMIA
Lymphohistiocytosis (LH) is a familial syndrome characterized by abnormal accumulation of non-malignant histiocytes in lymph nodes, liver, and spleen, erythrophagocytosis, hepatoaplenomegaly, recurrent fever, ...
