01. Gene therapy Boulikas.pdf - Gene therapy & Molecular Biology

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109. Gene Therapy /Phase I /Monogenic Disease /Chronic Granulomatous Disease 110. Gene Therapy /Phase I /II /Cancer /Colorectal Carcinoma Expressing TAG-72 111. Gene Therapy /Phase I /Cancer /Melanoma /Breast /Head and Neck Cancer /Cutaneous T- Cell Lymphoma /Immumotherapy 112. Gene Therapy /Phase I /Monogenic Disease /Leukocyte Adherence Deficiency (LAD) 113. Gene Therapy /Phase I /II /Cancer /Prostate /Immunotherapy 114. Gene Therapy /Phase I /II /Cancer /Chronic Myelogenous Leukemia /Adoptive Immunotherapy 115. Gene Therapy /Phase I /Cancer /Mesothelioma /Pro-Drug 116. Gene Therapy /Phase II /Infectious Disease /Human Immunodeficiency Virus 117. Gene Therapy /Phase II /Infectious Disease /Human Immunodeficiency Virus /Replication Inhibition /Hammerhead Ribozyme 118. ADENOVIRUS Gene Therapy /Phase I /Monogenic Disease /Cystic Fibrosis 119. Gene Therapy /Phase I /Monogenic Disease /Cystic Fibrosis Boulikas: An overview on gene therapy Fibronectin-Assisted, Retroviral-Mediated Transduction of CD34 + Peripheral Blood Cells with gp91 phox in Patients with X-Linked Chronic Granulomatous Disease: A Phase I Study A Phase I /II Study of Autologous CC49-Zeta Gene- Modified T Cells and a-Interferon in Patients with Advanced Colorectal Carcinomas Expressing the Tumor-Associated Antigen, TAG-72 Sponsor: Cell Genesys, Inc. IL-12 Gene Therapy Using Direct Injection of Tumors with Genetically Engineered Autologous Fibroblasts Retrovirus-Mediated Transfer of the cDNA for Human CD18 into Perpheral Blood Repopulating Cells of Patients with Leukocyte Adherence Deficiency Phase I /II Study of Allogeneic Human GM-CSF Gene Transduced Irradiated Prostate Cancer Cell Vaccines in Patients with Prostate Cancer Infusion of Polyclonal HyTK (hygromycin phosphotransferase and HSV thymidine kinase gene)transduced Donor T Cells for Adoptive Immunotherapy in Patients with Relapsed CML after Allogeneic Stem Cell Transplant: Phase I-II Clinical Trial The Treatment of Malignant Pleural Mesothelioma with aGene-Modified Cancer Vaccine: A Phase I Study A Phase II Study of Autologous CD4-Zeta Gene- Modified T Cells in HIV-Infected Patients with Undectable Plasma Viremia on Combination Antiretroviral Drug Therapy Sponsor: Cell Genesys, Inc. High Dose Chemotherapy and Autologous Peripheral Stem Cell Transplantation for HIV Lymphomas: A Phase IIa Study of Comparative Marking Using a Ribozyme Gene and a Neutral Gene Sponsor: Ribozyme Pharmaceuticals, Inc. A Phase I Study, in Cystic Fibrosis Patients, of the Safety, Toxicity, and Biological Efficacy of a Single Administration of a Replication Deficient, Recombinant Adenovirus Carrying the cDNA of the Normal Human Cystic Fibrosis Transmembrane Conductance Regulator Gene in the Lung. Gene Therapy of Cystic Fibrosis Lung Diseases Using E1 Deleted Adenoviruses: A Phase I Trial. 174 In Vitro /G-CSF Mobilized CD34 + Autologous Peripheral Blood Cells /Retrovirus /gp91phox /Intravenous Infusion In Vitro /Autologous CD8 + and CD4 + T Lymphocytes /Retrovirus /CC49-Zeta T Cell Receptor /Intravenous Infusion In Vitro /Autologous Fibroblasts /Lethally Irradiated /Retrovirus /Cytokine /Interleukin-12 /Intratumoral Injection In Vitro /G-CSF Mobilized CD34 + Autologous Peripheral Blood Cells /Retrovirus /CD18 /Intravenous Infusion In Vitro /Allogeneic Tumor Cells /Lethally Irradiated /Retrovirus /Cytokine /Granulocyte- Macrophage Colony Stimulating Factor /Subcutaneous Injection In Vitro /Donor CD8 + and CD4 + Lymphocytes /Retrovirus /Hygromycin Phosphotransferase- Herpes Simplex Thymidine Kinase Fusion Gene /Intravenous Infusion In Vivo /Allogeneic Tumor Cells /Lethally Irradiated /Retrovirus /Herpes Simplex Virus Thymidine Kinase /Ganciclovir /Intrapleural Administration In Vitro /Autologous CD8 + T Cells /Retrovirus /CD4-Zeta Chimeric Receptor /Intravenous In Vitro /CD34 + Autologous Peripheral Blood Cells /Retrovirus /Tat and Rev Hammerhead Ribozyme /Intravenous In Vivo /Nasal Epithelial Cells /Respiratory Epithelial Cells /Adenovirus /Serotype 5 /Cystic Fibrosis Transmembrane Conductance Regulator cDNA /Intranasal /Respiratory Tract Administration (Bronchoscope) In Vivo /Nasal Epithelial Cells /Respiratory Epithelial Cells /Adenovirus /Serotype 5 /E2a Temperature Sensitive Mutant /Cystic Fibrosis Transmembrane Conductance Regulator cDNA /Intranasal /Respiratory Tract Administration (Bronchoscope) Smith, Franklin O., and Dinauer, Mary C Venook, Alan Park, Chan H Hickstein, Dennis Simons, Jonathan W Flowers, Mary E. D. and Riddell, Stanley Schwarzenberge r, Paul Deeks, Steven G Krishnan, Amrita and Zaia, John, A Crystal, Ronald G Wilson, James M
120. Gene Therapy /Phase I /Monogenic Disease /Cystic Fibrosis 121. Gene Therapy /Phase I /Monogenic Disease /Cystic Fibrosis 122. Gene Therapy /Phase I /Monogenic Disease /Cystic Fibrosis 123. Gene Therapy /Phase I /Monogenic Disease /Cystic Fibrosis 124. Gene Therapy /Phase I /Cancer /Non-small Cell Lung Cancer /Tumor Suppressor Gene 125. Gene Therapy /Phase I /Monogenic Disease /Cystic Fibrosis 126. Gene Therapy /Phase I /Cancer /Central Nervous System /Pro- Drug 127. Gene Therapy /Phase I /Cancer /n /Pro-Drug 128. Gene Therapy /Phase I /Monogenic Disease /Cystic Fibrosis 129. Gene Therapy /Phase I /Monogenic Disease /Cystic Fibrosis 130. Gene Therapy /Phase I /Cancer /Head and Neck Squamous Cell /Tumor Suppressor Gene 131. Gene Therapy /Phase I /Cancer /Colon /Hepatic Metastases /Tumor Suppressor Gene 132. Gene Therapy /Phase I /Cancer /Central Nervous System Malignancies /Pro- Drug Gene Therapy and Molecular Biology Vol 1, page 175 Cystic Fibrosis Gene Therapy Using an Adenovirus Vector: In Vivo Safety and Efficacy in Nasal Epithelium. Sponsor: Genzyme Corporation A Phase I Study of Gene Therapy of Cystic Fibrosis Utilizing a Replication Deficient Recombinant Adenovirus Vector to Deliver the Human Cystic Fibrosis Transmembrane Conductance Regulator cDNA to the Airways. Sponsor: Genetic Therapy, Inc. /Novartis Gene Therapy for Cystic Fibrosis Using E1 Deleted Adenovirus: A Phase I Trial in the Nasal Cavity. Adenovirus-Mediated Gene Transfer of CFTR to the Nasal Epithelium and Maxillary Sinus of Patients with Cystic Fibrosis. Sponsor: Genzyme Corporation Clinical Protocol for Modification of Tumor Suppressor Gene Expression and Induction of Apoptosis in Non-Small Cell Lung Cancer (NSCLC) with an Adenovirus Vector Expressing Wildtype p53 and Cisplatin. Evaluation of Repeat Administration of a Replication Deficient, Recombinant Adenovirus Containing the Normal Cystic Fibrosis Transmembrane Conductance Regulator cDNA to the Airways of Individuals with Cystic Fibrosis. Treatment of Advanced CNS Malignancy with the Recombinant Adenovirus H5.020RSVTK: A Phase I Trial. Treatment of Advanced Mesothelioma with the Recombinant Adenovirus H5.010RSVTK: A Phase I Trial. Adenovirus Mediated Gene Transfer for Cystic Fibrosis: Safety of Single Administration in the Lung (lobar instillation). Sponsor: Genzyme Corporation Adenovirus Mediated Gene Transfer for Cystic Fibrosis: Safety of a Single Administration in the Lung (aerosol administration). Sponsor: Genzyme Corporation Clinical Protocol for Modification of Tumor Suppressor Gene Expression in Head and Neck Squamous Cell Carcinoma (HNSCC) with an Adenovirus Vector Expressing Wild-type p53. Gene Therapy of Primary and Metastatic Malignant Tumors of the Liver Using ACN53 Via Hepatic Artery Infusion: A Phase I Study. Sponsor: Schering Plough Corporation (formerly Canji) Phase I Study of Adenoviral Vector Delivery of the HSV-TK Gene and the Intravenous Administration of Ganciclovir in Adults with Malignant Tumors of the Central Nervous System. 175 In Vivo /Nasal Epithelial Cells /Adenovirus /Serotype 2 /Cystic Fibrosis Transmembrane Conductance Regulator cDNA /Intranasal In Vivo /Nasal Epithelial Cells /Respiratory Epithelial Cells /Adenovirus /Serotype 5 /Cystic Fibrosis Transmembrane Conductance Regulator cDNA /Intranasal /Respiratory Tract Administration (Bronchoscope) In Vivo /Nasal Epithelial Cells /Adenovirus /Serotype 5 /Cystic Fibrosis Transmembrane Conductance Regulator CDNA /Intranasal In Vivo /Nasal Epithelial Cells /Maxillary Sinus Epithelial Cells /Adenovirus /Serotype 2 /Cystic Fibrosis Transmembrane Conductance Regulator cDNA /Intranasal /Maxillary Sinus Administration In Vivo /Autologous Tumor Cells /Adenovirus /Serotype 5 /p53 cDNA /Intratumoral /Bronchoscope In Vivo /Nasal Epithelial Cells /Respiratory Epithelial Cells /Adenovirus /Serotype 5 /Cystic Fibrosis Transmembrane Conductance Regulator cDNA /Intranasal /Respiratory Tract Administration (Bronchoscope) /Multiple Dose In Vivo /Autologous Tumor Cells /Adenovirus /Serotype 5 /Herpes Simplex Virus Thymidine Kinase cDNA /Ganciclovir /Stereotactic Injection In Vivo /Autologous Tumor Cells /Adenovirus /Serotype 5 /Herpes Simplex Virus Thymidine Kinase cDNA /Ganciclovir /Intrapleural In Vivo /Respiratory Epithelial Cells /Adenovirus /Serotype 2 /Cystic Fibrosis Transmembrane Conductance Regulator cDNA /Respiratory Epithelial Cells /Bronchoscope In Vivo /Respiratory Epithelial Cells /Adenovirus /Serotype 2 /Cystic Fibrosis Transmembrane Conductance Regulator cDNA /Respiratory Epithelial Cells /Aerosol Administration In Vivo /Autologous Tumor Cells /Adenovirus /Serotype 5 /p53 cDNA /Intratumoral /Bronchoscope In Vivo /Autologous Tumor Cells /Adenovirus /Serotype 5 /p53 cDNA /Intrahepatic /Hepatic Artery /Bolus Infusion In Vivo /Autologous Tumor Cells /Adenovirus /Serotype 5 /Herpes Simplex Virus Thymidine Kinase cDNA /Ganciclovir /Intra- tumoral /Stereotactic Injection Welsh, Michael J and Smith, Alan E Wilmott, Robert W Boucher, Richard C. and Knowles, Michael R Welsh, Michael J Roth, Jack A Crystal, Ronald G Eck, Stephen L. and Alavi, Jane B Albelda, Steven M Dorkin, Henry L and Lapey, Allen Dorkin, Henry L. and Lapey, Allen Clayman, Gary Venook, Alan and Warren, Robert Grossman, Robert and Woo, Savio
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Gene Ther Mol Biol Vol 1, 1-172. Ma
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I. Introduction Monumental progress
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The use of retroviral vectors in hu
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displaying the cleavable EGF domain
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molecules in the nucleus (Lowe and
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sensitive mutations which allow pro
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processed as described in panel A s
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On the contrary, the payload capaci
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in K562 human erythroleukemia cells
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defective HSV virus (DeLuca and Sch
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complex into the cytosol from the e
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Gene Therapy and Molecular Biology
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delivered by Sendai virus-liposome
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plasmids with the cell surface, tra
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infected by adenovirus alone; infec
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B. Transcription factor binding sit
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A closely related family of ubiquit
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cells. I-CreI is a member of this c
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C. Considerations of chromatin stru
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A. The molecular basis of cancer im
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fragments, or heat shock proteins c
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ecombinant vaccinia virus expressin
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HIV-1 envelope DNA construct develo
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inding sites A GYYY oriented in opp
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Prives, 1994). Whereas the wild-typ
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mutant p53 (mutations on p53 are wi
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master regulator of the cell cycle
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Work from several groups has shown
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chromatin condensation, drop in pH,
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Figure 23. A pathway leading to the
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Excessive necrotic death in cells o
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implantation, and similar processes
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normal cells in the surroundings. T
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A bicistronic retroviral vector (Ha
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C. Antisense ras gene transfer for
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equal to 6.0 showed S1 hyperreactiv
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protein (e.g. Smith et al, 1995; Fo
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transduced primary mouse fibroblast
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IL-1 - receptor antagonist protein
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p53 lung cancer retroviru s MHC cla
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designed by immunization with porti
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Isolation of an RNA aptamer that ca
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induction of human apoE in neonate
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atrium in heart, endothelial cells
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which eliminated tumors in small an
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C. Transfer of other genes against
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significant reduction in neointima
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(Prehn et al, 1996); this implies a
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B. Approaches to gene therapy of RA
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of human immunoglobulin and antigen
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A. Etiology and mechanisms of destr
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However, CsA also inhibits the acti
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Page 127 and 128: Introgen Therapeutics (Austin and H
Page 129 and 130: When a subfragment of only 513 bp o
Page 131 and 132: Armentano D, Zabner J, Sacks C, Soo
Page 133 and 134: Brady HJM, Miles CG, Pennington DJ,
Page 135 and 136: Chen J, Stickles RJ, Daichendt KA (
Page 137 and 138: Day ML, Wu S, Basler JW (1993) Pros
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Page 141 and 142: Giovannangeli C, Perrouault L, Escu
Page 143 and 144: the neoplastic phenotype by replace
Page 145 and 146: integrate in a site-specific fashio
Page 147 and 148: Li R, Waga S, Hannon GJ, Beach D, S
Page 149 and 150: adiation-induced apoptosis in the g
Page 151 and 152: Nakaya T, Iwai S, Fujinaga K, Sato
Page 153 and 154: Polyak K, Xia Y, Zweier JL, Kinzler
Page 155 and 156: macrophages from simian immunodefic
Page 157 and 158: intimal hyperplasia in a rat caroti
Page 159 and 160: Trono D, Feinberg MB, Baltimore D (
Page 161 and 162: Wattiaux R, Jadot M, Warnier-Pirott
Page 163 and 164: similar to mammalian interleukin-1
Page 165 and 166: Gene Therapy and Molecular Biology
Page 167 and 168: 24. Gene Marking /Infectious Diseas
Page 169 and 170: Drug 50. Gene Therapy /Phase I /Can
Page 171 and 172: 76. Gene Therapy /Phase I /Cancer /
Page 173: 99. Gene Therapy /Phase II /Cancer
Page 177 and 178: Gene Therapy and Molecular Biology
Page 179 and 180: cancer not specified) /Immunotherap
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