BOOK
Gene Therapy, An Issue of Hematology/Oncology Clinics of North America, E-Book
Daniel E. Bauer | Donald B Kohn
(2017)
Additional Information
Book Details
Abstract
This issue of Hematology/Oncology Clinics will focus on Gene Therapy. Topics include, but are not limited to Historical Perspective and Current Renaissance, Integrating Vectors, Nonintegrating Vectors, Gene Editing, Conditioning Therapies for Autologous HSCT, Approaches to Immunodeficiency, Approaches to Hemoglobinopathy, Approaches to Hemophilia, Hematopoietic Gene Therapies for Neurologic and Metabolic Disease, Gene Therapy Approaches to HIV and other Infectious Diseases, HSC Approaches to Cancer, and Gene Modified T Cell Therapies for Cancer.
Table of Contents
| Section Title | Page | Action | Price |
|---|---|---|---|
| Front Cover | Cover | ||
| HEMATOLOGY/ONCOLOGYCLINICS OF NORTH AMERICA | i | ||
| Copyright\r | ii | ||
| Contributors | iii | ||
| CONSULTING EDITORS | iii | ||
| EDITORS | iii | ||
| AUTHORS | iii | ||
| Contents | vii | ||
| Preface: Gene Therapy | vii | ||
| Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy | vii | ||
| Integrating Vectors for Gene Therapy and Clonal Tracking of Engineered Hematopoiesis | vii | ||
| Nonintegrating Gene Therapy Vectors | vii | ||
| In Vivo Hematopoietic Stem Cell Transduction | viii | ||
| Therapeutic Gene Editing Safety and Specificity | viii | ||
| Gene Editing: Regulatory and Translation to Clinic | viii | ||
| Opening Marrow Niches in Patients Undergoing Autologous Hematopoietic Stem Cell Gene Therapy | viii | ||
| Gene Therapy Approaches to Immunodeficiency | ix | ||
| Gene Therapy Approaches to Hemoglobinopathies | ix | ||
| Gene Therapy for Hemophilia | ix | ||
| Hematopoietic Gene Therapies for Metabolic and Neurologic Diseases | ix | ||
| Gene Therapy Approaches to Human Immunodeficiency Virus and Other Infectious Diseases | x | ||
| Hematopoietic Stem Cell Approaches to Cancer | x | ||
| Gene Modified T Cell Therapies for Hematological Malignancies | x | ||
| HEMATOLOGY/ONCOLOGY\rCLINICS OF NORTH AMERICA\r | xi | ||
| FORTHCOMING ISSUES | xi | ||
| December 2017 | xi | ||
| February 2018 | xi | ||
| April 2018 | xi | ||
| RECENT ISSUES | xi | ||
| August 2017 | xi | ||
| June 2017 | xi | ||
| April 2017 | xi | ||
| Preface:\rGene Therapy | xiii | ||
| Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy | 721 | ||
| Key points | 721 | ||
| IN THE BEGINNING | 722 | ||
| GAMMARETROVIRAL VECTORS EMERGE | 722 | ||
| RISE OF THE LENTIVIRAL VECTORS | 725 | ||
| THIS IS THE DAWNING OF THE AGE OF GENE EDITING | 727 | ||
| ONGOING CHALLENGES | 728 | ||
| REFERENCES | 730 | ||
| Integrating Vectors for Gene Therapy and Clonal Tracking of Engineered Hematopoiesis | 737 | ||
| Key points | 737 | ||
| INTRODUCTION | 738 | ||
| GAMMARETROVIRAL AND LENTIVIRAL VECTORS | 738 | ||
| TRANSPOSON-BASED VECTORS | 740 | ||
| CLINICAL TRIALS WITH INTEGRATING VECTORS AND FURTHER APPLICATIONS | 741 | ||
| INSERTIONAL MUTAGENESIS AS A DOUBLE-EDGED SWORD: AN IMPORTANT TOOL TO UNDERSTAND GENE FUNCTION AND NETWORKS BUT A GREAT CHA ... | 742 | ||
| SAFETY OF GENE THERAPY AND INTEGRATION SITE ANALYSIS | 742 | ||
| INSERTIONAL PROPERTIES OF RETROVIRAL VECTORS | 743 | ||
| METHODS AND EVOLUTION OF INSERTION SITES RETRIEVAL | 744 | ||
| USE OF INSERTIONAL TAGGING FOR CLONAL TRACKING STUDIES | 746 | ||
| SUMMARY | 747 | ||
| REFERENCES | 747 | ||
| Nonintegrating Gene Therapy Vectors | 753 | ||
| Key points | 753 | ||
| BRIEF HISTORICAL OVERVIEW OF GENE THERAPY | 754 | ||
| NONINTEGRATING VECTORS | 754 | ||
| NONINTEGRATING VIRAL VECTORS | 755 | ||
| Adenovirus Vectors | 755 | ||
| Future directions and hematopoietic application | 758 | ||
| Adeno-Associated Virus Vectors | 759 | ||
| Future directions and hematopoietic application | 759 | ||
| Integration-Deficient Lentiviral Vectors | 760 | ||
| Future directions and hematopoietic application | 761 | ||
| Poxviral Vectors, Including Vaccinia | 762 | ||
| Future directions and hematopoietic application | 763 | ||
| Other Nonintegrating Viral Vector Systems | 763 | ||
| Nonviral Vectors | 763 | ||
| Future directions and hematopoietic application | 765 | ||
| SUMMARY | 765 | ||
| ACKNOWLEDGMENTS | 766 | ||
| REFERENCES | 766 | ||
| In Vivo Hematopoietic Stem Cell Transduction | 771 | ||
| Key points | 771 | ||
| INTRODUCTION | 771 | ||
| Hematopoietic Stem Cells in the Bone Marrow | 771 | ||
| Hematopoietic Stem Cell Mobilization | 772 | ||
| EXAMPLES FOR IN VIVO HEMATOPOIETIC STEM CELL GENE THERAPY | 772 | ||
| Example 1: Intravenous Injection of Triplex-Forming Peptide Nucleic Acids | 772 | ||
| Example 2: Intravenous Injection of Foamy Virus Vectors in Dogs | 773 | ||
| Example 3: Intraosseal Lentivirus Injection in Mice | 775 | ||
| Example 4: Hematopoietic Stem Cell Transduction Using Adeno-Associated Virus Vectors | 776 | ||
| Example 5: In Vivo Transduction of Primitive Hematopoietic Stem Cells After Mobilization and Intravenous Injection of Integ ... | 777 | ||
| DISCUSSION | 779 | ||
| REFERENCES | 780 | ||
| Therapeutic Gene Editing Safety and Specificity | 787 | ||
| Key points | 787 | ||
| INTRODUCTION | 787 | ||
| SAFETY | 788 | ||
| SPECIFICITY | 791 | ||
| SUMMARY | 793 | ||
| ACKNOWLEDGMENT | 794 | ||
| REFERENCES | 794 | ||
| Gene Editing | 797 | ||
| Key points | 797 | ||
| INTRODUCTION | 797 | ||
| OVERVIEW OF ZINC FINGER NUCLEASES FOR GENOME EDITING | 798 | ||
| GENOME EDITING USING CCR5 ZINC FINGER NUCLEASE MRNA; MRNA PRODUCTION | 799 | ||
| CELL PURIFICATION AND MODIFICATION | 800 | ||
| HEMATOPOIETIC STEM AND PROGENITOR CELLS ADMINISTRATION TO HUMAN SUBJECTS | 800 | ||
| REGULATORY STRATEGY FOR PRECLINICAL DEVELOPMENT | 801 | ||
| KNOCK-OUT | 801 | ||
| CCR5 ZINC FINGER NUCLEASE ACTIVITY | 801 | ||
| IN VITRO AND IN VIVO STEM CELL FUNCTIONAL ASSAYS | 802 | ||
| CCR5 ZINC FINGER NUCLEASE ACTIVITY | 802 | ||
| ASSESSING POTENTIAL GENOTOXICITY OF CCR5 ZINC FINGER NUCLEASE | 803 | ||
| Opening Marrow Niches in Patients Undergoing Autologous Hematopoietic Stem Cell Gene Therapy | 809 | ||
| Key points | 809 | ||
| INTRODUCTION | 809 | ||
| CONDITIONING PATIENTS BEFORE AUTOLOGOUS GENE-CORRECTED HEMATOPOIETIC CELL TRANSPLANTATION | 811 | ||
| CHEMOTHERAPEUTIC AGENTS USED AS PRETREATMENT IN HEMATOPOIETIC CELL GENE THERAPY | 813 | ||
| Busulfan | 814 | ||
| Melphalan | 815 | ||
| Thiotepa | 815 | ||
| Treosulfan | 816 | ||
| NONCHEMOTHERAPY APPROACHES TO CONDITIONING BEFORE INFUSION OF GENE-MODIFIED HEMATOPOIETIC STEM CELLS | 816 | ||
| CD45 | 816 | ||
| CD117 | 818 | ||
| SUMMARY | 819 | ||
| REFERENCES | 819 | ||
| Gene Therapy Approaches to Immunodeficiency | 823 | ||
| Key points | 823 | ||
| INTRODUCTION | 823 | ||
| ADENOSINE DEAMINASE DEFICIENCY | 824 | ||
| X-LINKED SEVERE COMBINED IMMUNODEFICIENCY | 825 | ||
| CHRONIC GRANULOMATOUS DISEASE | 826 | ||
| WISKOTT-ALDRICH SYNDROME | 827 | ||
| PRECLINICAL APPROACHES IN OTHER PRIMARY IMMUNE DISORDERS | 828 | ||
| SUMMARY | 830 | ||
| REFERENCES | 830 | ||
| Gene Therapy Approaches to Hemoglobinopathies | 835 | ||
| Key points | 835 | ||
| INTRODUCTION | 835 | ||
| DEVELOPING GENE TRANSFER VECTORS FOR HEMOGLOBINOPATHIES | 837 | ||
| GENE THERAPY FOR β-THALASSEMIA: RATIONALE AND REQUIREMENTS | 837 | ||
| GENE THERAPY FOR β-THALASSEMIA: PRECLINICAL STUDIES | 839 | ||
| CLINICAL GENE THERAPY FOR β-THALASSEMIA | 841 | ||
| GENE THERAPY FOR SICKLE CELL DISEASE: CHALLENGES AND INITIAL STUDIES | 844 | ||
| CLINICAL GENE THERAPY FOR SICKLE CELL DISEASE | 845 | ||
| GENE THERAPY FOR HEMOGLOBINOPATHIES: FUTURE DIRECTIONS | 846 | ||
| SUMMARY | 847 | ||
| REFERENCES | 847 | ||
| Gene Therapy for Hemophilia | 853 | ||
| Key points | 853 | ||
| INTRODUCTION | 853 | ||
| FIRST CLINICAL STUDIES OF GENE THERAPY IN HEMOPHILIA | 855 | ||
| CURRENT AND ON-GOING TRIALS OF GENE THERAPY FOR HEMOPHILIA A AND B | 856 | ||
| THE FIRST LONG-TERM SUCCESS IN A CLINICAL TRIAL OF GENE TRANSFER IN HEMOPHILIA | 862 | ||
| RECENT TRIALS OF GENE TRANSFER IN HEMOPHILIA B | 863 | ||
| ADENO-ASSOCIATED VIRAL VECTORS AND GENE THERAPY FOR HEMOPHILIA A | 863 | ||
| OBSTACLES TO WIDER USE OF ADENO-ASSOCIATED VIRAL VECTOR TECHNOLOGY | 864 | ||
| Safety Considerations | 864 | ||
| Scale-Up of Vector Production | 865 | ||
| AFFORDABILITY OF GENE THERAPY | 865 | ||
| SUMMARY | 865 | ||
| REFERENCES | 866 | ||
| Hematopoietic Gene Therapies for Metabolic and Neurologic Diseases | 869 | ||
| Key points | 869 | ||
| INTRODUCTION | 869 | ||
| MICROGLIA | 870 | ||
| Microglia in Neurodegenerative Diseases | 870 | ||
| Microglia and metabolic storage disorders | 870 | ||
| Microglia and other neurodegenerative diseases | 871 | ||
| Microglia Origin in the Developing Brain | 873 | ||
| Microglia Maintenance in Adulthood | 873 | ||
| REPLACING MICROGLIA BY HEMATOPOIETIC STEM CELL TRANSPLANTATION: THE CASE OF STORAGE DISORDERS | 874 | ||
| Hematopoietic Stem Cell Gene Therapy: Improving Safety and Enhancing Benefits of Allogeneic Hematopoietic Cell Transplantation | 875 | ||
| REFERENCES | 877 | ||
| Gene Therapy Approaches to Human Immunodeficiency Virus and Other Infectious Diseases | 883 | ||
| Key points | 883 | ||
| INTRODUCTION | 883 | ||
| GENE EDITING: ENGINEERING RESISTANCE TO VIRUSES | 884 | ||
| ELIMINATING VIRAL ENTRY RECEPTORS | 884 | ||
| DIRECTLY TARGETING VIRAL GENOMES | 886 | ||
| SYSTEMIC EXPRESSION OF ANTIVIRAL FACTORS | 887 | ||
| INTRODUCING OTHER VIRAL RESISTANCE FACTORS | 888 | ||
| ENGINEERING IMMUNITY | 889 | ||
| ANTIBACTERIAL STRATEGIES | 889 | ||
| SUMMARY | 889 | ||
| REFERENCES | 890 | ||
| Hematopoietic Stem Cell Approaches to Cancer | 897 | ||
| Key points | 897 | ||
| INTRODUCTION | 897 | ||
| Hematopoietic Stem Cells | 897 | ||
| Improving Engraftment of Hematopoietic Stem Cells | 899 | ||
| Expansion of hematopoietic stem cells | 899 | ||
| Selection of gene-edited and chemoprotected hematopoietic stem cells in vivo | 900 | ||
| Examples of Hematopoietic Stem Cell Gene Therapy for Cancer | 901 | ||
| MGMTP140K-resistant blood cells and cancer therapies: Glioblastoma | 901 | ||
| Hematopoietic stem cells and immunotherapy | 906 | ||
| SUMMARY | 907 | ||
| REFERENCES | 908 | ||
| Gene Modified T Cell Therapies for Hematological Malignancies | 913 | ||
| Key points | 913 | ||
| INTRODUCTION | 913 | ||
| GENETIC REDIRECTION OF T CELLS AGAINST TUMOR ANTIGENS | 913 | ||
| GENE-EDITING ANTILEUKEMIC T CELLS | 915 | ||
| MANUFACTURING, DISSEMINATION, AND COMMERCIALIZATION | 918 | ||
| REFERENCES | 922 |