AN INTRODUCTION TO MOLECULAR MEDICINE AND GENE THERAPY
CONTENTS CHAPTER MOLECULAR MEDICINE AND GENE THERAPY: AN INTRODUCTION INTRODUCTION GENETIC MANIFESTATIONS OF MOLECULAR MEDICINE GENE THERAPY AND PATTERNS OF GENE EXPRESSION GENE THERAPY AND MOLECULAR MEDICINE GENE THERAPY: CURRENT BASIC SCIENCE ISSUES HUMAN GENE THERAPY: CURRENT STATUS AND BASIC SCIENCE RESEARCH NEEDS GENE THERAPIES: NEXT HORIZON KEY CONCEPTS SUGGESTED READINGS CHAPTER NUCLEAR TRANSPLANTATION AND NEW FRONTIERS IN GENETIC MOLECULAR MEDICINE BACKGROUND NUCLEAR TRANSPLANTATION: A TOOL IN DEVELOPMENTAL BIOLOGY TECHNICAL DEVELOPMENTS IN NUCLEAR TRANSPLANTATION DE?NING THE LIMITS OF NUCLEAR REPROGRAMMING IN MAMMALS TOWARD AN UNDERSTANDING OF THE MECHANISMS OF GENETIC REPROGRAMMING APPLICATION OF GENETIC REPROGRAMMING HUMAN EMBROYONIC STEM CELL RESEARCH: AN ETHICS NOTE SUMMARY BUILING A BETTER MOUSE: GENETICALLY ALTERED MICE AS MODELS FOR GENE THERAPY PRODUCING MOUSE MODELS OF HUMAN DISEASE MOUSE MODELS FOR GENE THERAPY: WHAT MAKES A GOOD MODEL OF HUMAN DISEASE? MODELS OF MONOGENIC DISORDERS MODELS OF POLYGENIC AND MULTIFACTORIAL DISORDERS MOUSE MODELS OF MOLECULAR THERAPEUTICS: DEVELOPING AND TESTING GENE THERAPY METHODOLOGY GENERATION OF CHIMERIC TISSUES HUMAN CELL XENOGRAFT MODELS IN IMMUNODE?CIENT MICE MOUSE MODELS: THE NEXT GENERATION VECTORS IN GENE THERAPY VIRAL VECTORS USED FOR GENE THERAPY RETROVIRAL VECTORS ADENOVIRAL VECTORS ADENOVIRUS-ASSOCIATED VIRUS HERPES SIMPLEX VIRUS OTHER VIRAL VECTORS NONVIRAL VECTORS OLIGONUCLEOTIDES GENE TARGETING BACKGROUND AND CHALLENGES INTRODUCTION OF DNA INTO THE CELL NONVIRAL TRANSFER VEHICLES RECOMBINATIONAL AND REPAIR ENZYMES IN GENE TARGETING EFFORTS SYNTHETIC OLIOGONUCLEOTIDES AS TOOLS FOR TARGETING INSERTION OF FRAGMENTS OF DNA: GENE DISRUPTION AND REPLACEMENT GENE TARGETING HAS ALREADY PROVEN USEFUL GENE TARGETING: THE FUTURE GENE THERAPY FOR HEMATOLOGICAL DISORDERS REQUIREMENTS FOR GENE TRANSFER INTO HEMATOPOIETIC CELLS HEMATOPOIETIC STEM AND PROGENITOR CELLS AS TARGETS FOR GENE THERAPY LYMPHOCYTE GENE TRANSFER CURRENT PROBLEMS AND FUTURE DIRECTIONS GENE THERAPY FOR LIVER DISEASE GENERAL PRINCIPLES FOR HEPATIC GENE THERAPY CLINICAL APPLICATIONS OF LIVER-DIRECTED GENE THERAPY GENE THERAPY IN CARDIOVASCULAR DISEASE GENETIC MANIPULATION OF CARDIOVASCULAR TISSUE GENE THERAPY OF RESTENOSIS GENE THERAPY FOR ANGIOGENESIS GENE THERAPY OF VASCULAR GRAFTS GENE THERAPY FOR THE HEART COMPONENTS OF CELL AND GENE THERAPY FOR NEUROLOGICAL DISORDERS SORTING OUT THE COMPLEXITY OF THE NERVOUS SYSTEM WHAT GOES WRONG IN NEUROLOGICAL DISORDERS? NEUROTOPHIC FACTORS AND GENE THERAPY NEURAL TRANSPLANTS AND STEM CELLS CLINICAL NEURODEGENERATIVE CONDITIONS CLINICAL TRIALS TESTING GENETICALLY MODI?ED CELLS AND NEUROTROPHIC FACTORS FOR NEURODEGENERATION FUTURE CONSIDERATIONS AND ISSUES GENE THERAPY IN THE TREATMENT OF CANCER GENETIC BASIS OF CARCINOGENESIS GENE THERAPY APPROACHES TO THE TREATMENT OF CANCER DNA CANCER VACCINES GENE THERAPY FOR HIV INFECTION GENETIC ORGANIZATION OF HIV LIFE CYCLE AND PATHOGENESIS OF HIV-1 INFECTION GENETIC APPROACHES TO INHIBIT HIV REPLICATION TRANSDOMINANT NEGATIVE PROTEINS NUCLEIC-ACID-BASED GENE THERAPY APPROACHES GENETIC APPROACHES TO ENHANCE IMMUNITY IN AIDS STIMULATION OF AN HIV-SPECI?C IMMUNE RESPONSE PRACTICAL ASPECTS OF GENE THERAPY FOR HIV ANTI-HIV GENE THERAPY CLINICAL TRIALS CONCLUSIONS GENE THERAPY APPROACHES FOR RHEUMATOID ARTHRITIS GENERAL CONSIDERATIONS ABOUT GENE THERAPY IN RA DELIVERY OF GENE TO SYNOVIAL CELLS ANIMAL MODELS TO TEST GENE THERAPY APPROACHES CURRENT TARGETS FOR GENE THERAPY IN RA CLINICAL STATUS AND FUTURE PERSPECTIVE FEDERAL OVERSIGHT OF GENE THERAPY RESEARCH OF?CE OF BIOTECHNOLOGY ACTIVITIES RECOMBINANT DNA ADVISORY COMMITTEE CURRENT REVIEW AND APPROVAL PROCESS MOVING TOO FAST IN A FIELD OF PROMISE INSTITUTIONAL REVIEW OF RECOMBINANT DNA RESEARCH INTERNATIONAL EFFORTS AT REGULATORY OVERSIGHT ETHICAL ISSUES IN MOLECULAR MEDICINE AND GENE THERAPY MOLECULAR MEDICINE AND GENE THERAPY: ETHICAL ISSUES IN THE CLINICAL CONTEXT GENE THERAPY: ETHICAL ISSUES AT THE POLICY LEVEL EPILOGUE: PERSONAL GENETIC MEDICINE—THE FUTURE IS NOW DNA DATABASES INDIVIDUAL GENETIC PREVENTIVE MEDICINE INDIVIDUAL GENETIC MEDICINE AND PHARMACOTHERAPY