Textbook of gene therapy:
Gespeichert in:
| 1. Verfasser: | |
|---|---|
| Format: | Buch |
| Sprache: | English |
| Veröffentlicht: |
Seattle [u.a.]
Hogrefe & Huber
1998
|
| Schlagworte: | |
| Online-Zugang: | Inhaltsverzeichnis |
| Beschreibung: | XVIII, 434 S. Ill. |
| ISBN: | 0889371903 |
Internformat
MARC
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Datensatz im Suchindex
| _version_ | 1804126179790159872 |
|---|---|
| adam_text | Contents
Foreword by Patrick Aebischer v
Preface vii
Abbreviations xv
Glossary xvii
1 Introduction and Historical Aspects 1
Definitions 1
Historical Development 1
Classification 1
Relation of Gene Therapy to Genomics and Biotechnology 1
2 Molecular Biological Basics for Gene Therapy 5
DNA 5
RNA 7
Genes 9
Genome 11
The Human Genome Project 11
Chromosomes 12
Mitochondrial DNA 13
Polymerase Chain Reaction (PCR) 13
Cloning 14
3 Techniques of Gene Therapy 15
Introduction 15
Physical Methods of Gene Transfer 16
Chemical Methods of Gene Transfer 18
Gene Repair and Replacement 19
Cell Mediated Gene Therapy 19
Transplantation of Cells 23
Implantation of Genetically Engineered Cells 24
Innovative Methods for Gene Therapy 26
Germline Gene Therapy 31
Gene Marking 32
Methods of Assessing Gene Expression 33
4 Vectors for Gene Therapy 35
Introduction 35
The Ideal Vector for Gene Therapy 36
Viral Vectors 36
Nonviral Vectors for Gene Therapy 50
Concluding Remarks about Vectors 50
5 Routes of Administration and Target Tissues for Gene Therapy 61
Various Routes of Introduction of DNA into the Living Body 61
Direct Injection of Naked DNA 61
x Table of Contents
Direct Injection of DNA Conjugated to Liposomes Carrier 61
Intravenous DNA Injection 62
Intra Arterial Delivery 62
Particle Mediated Transfer to Various Tissues and Lesions by Gene Gun 62
Target Organs and Tissues for Gene Therapy 63
6 Antisense Therapy 73
Introduction 73
Molecular Basis of Antisense Therapeutics 73
Pharmacology 75
Methods of Delivery 76
Triplex Formation 78
Ribozymes 80
Clinical Applications of Antisense Oligonucleotides 83
Advantages and Limitations of Antisense Approach 91
Concluding Remarks and Future Potential 91
7 Immunogene Therapy 93
Introduction 93
DNA Based Vaccines 93
DNA Inoculation Technology 93
Vaccine Vectors 94
Methods for Enhancing the Potency of DNA Vaccines 94
Applications of DNA Vaccination 95
DNA Vaccine for Tuberculosis 95
DNA Vaccine for Malaria 95
DNA Vaccine for Leishmaniasis 96
Cancer Vaccines 96
Genetic Immunization Against Allergen Induced Disorders 96
DNA Immunization for Autoimmune Diseases 97
Antibody Based Gene Therapies 97
Intrabodies (Intracellular Antibodies) 97
Adverse Effects of DNA Vaccination 98
Commercial Development of DNA Vaccines 98
Future Prospects of DNA Vaccines 98
8 Animal Models for Evaluating Gene Therapy 101
Introduction 101
Transgenic Animals 101
Homologous Recombination 102
Animal Models of Human Diseases 102
Animal Models of Human Disease for Gene Therapy 104
9 Gene Therapy for Genetic Disorders 105
Introduction 105
Primary Immunodeficiency Disorders 106
Metabolic Disorders 109
Lysosomal Storage Diseases (LSD) 112
10 Gene Therapy for Cancer 117
Introduction 117
Molecular Biology of Cancer 117
Gene Transfer Techniques as Applied to Cancer Gene Therapy 123
Strategies for Cancer Gene Therapy 124
Hematopoietic Malignancies 143
Table of Contents xi
Malignant Melanoma 147
Cancer of the Colon 150
Gastric Cancer 151
Cancer of the Lung 151
Cancer of the Prostate 153
Ovarian Cancer 154
Cancer of the Breast 155
Head and Neck Tumors 157
Hepatocellular Carcinoma 157
Carcinoma of the Bladder 158
Renal Cancer 158
Pancreatic Cancer 159
Tumor Metastases 159
11 Gene Therapy for Malignant Brain T imors . 161
Introduction 161
Biology of Malignant Primary Brain Tumors 161
Diagnosis of Brain Tumors 162
Conventional Treatment of Malignant Brain Tumors 162
Emerging Therapies for Glioblastoma Multiforme 164
Gene Therapy for Glioblastoma Multiforme 165
Concluding Remarks 178
12 Gene Therapy for Neurological Disorders 179
Introduction 179
Special Techniques of Gene Therapy for the Nervous System 180
Degenerative Neurological Disorders 186
Cerebrovascular Disorders 211
CNS trauma 220
Injuries of the Spinal Cord 223
Neurogenetic Disorders 225
Epilepsy 228
Mitochondrial disorders 229
13 Gene Therapy for Cardiovascular Disorders 233
Introduction 233
Techniques of Gene Transfer to the Cardiovascular System 233
Angiogenesis and Gene Therapy of Ischemic Disorders 235
Genetic Cardiovascular Disorders 236
Acquired Cardiovascular Disease 238
Peripheral Arterial Disease 240
Concluding Remarks •.. ..... 246
14 Gene Therapy for Viral Infections 247
Introduction 247
AIDS 247
Gene Therapy in HIV Infection 249
Cytomegalovirus Infections •........• 257
DNA Vaccines for Other Viral Infections 257
Antisense Approach to Combating Miscellaneous Viral Infections 258
15 Gene Therapy for Hematological Disorders ........... 259
Introduction 259
Hemophilias ¦........• 259
Hemoglobinopathies 261
xii Table of Contents
Acquired Disorders 263
16 Gene Therapy for Liver Disorders 265
Introduction 265
Techniques of Gene Delivery to the Liver 265
Gene Therapy of Syndromes Affecting the Liver 267
17 Gene Therapy Endocrine and Metabolic Disorders 269
Introduction 269
Diabetes Mellitus 269
Growth Hormone Deficiency 270
Obesity 271
18 Gene Therapy for Pulmonary Disorders 273
Techniques of Gene Delivery to the Lungs 273
Cystic Fibrosis 274
Gene Therapy for CF 275
Miscellaneous Pulmonary Disorders 278
19 Gene Therapy for Diseases of Bones and Joints 281
Bone Fractures 281
Osteogenesis Imperfecta (01) 282
Rheumatoid Arthritis 282
20 Gene Therapy for Diseases of Skin and Soft Tissues 285
Gene Transfer to the Skin 285
Gene Therapy for Hair Loss 285
Gene Therapy for Wound Healing 285
Treatment of Various Skin Disorders 286
21 Gene Therapy for Gastrointestinal Disorders 289
Introduction 289
Methods of Gene Transfer to the Gastrointestinal Tract 289
Indications for Gastrointestinal Gene Therapy 290
Gene Therapy for Inflammatory Disorders of the Bowel 290
Gene Transfer to the Salivary Glands 290
22 Gene Therapy for Renal Disorders 293
Introduction 293
Methods of Gene Delivery to the Kidney 293
Gene Therapy in Animal Experimental Models of Renal Disease 295
Genetic Manipulations of the Embryonic Kidney 295
Therapeutic Intervention in Glomerulonephritis by Oligonucleotides (ODNs) .... 295
Gene Therapy for Renal Fibrosis 296
Use of Genetically Engineered Cells for the Treatment of Uremia Due to Renal Failure 296
23 Gene Therapy and Transplantation Medicine 297
Introduction 297
Bone Marrow Transplantation (BMT) 297
Gene Therapy for Graft Versus Host Disease Associated with BMT 298
Gene Therapy in Lung Transplantation 298
Gene Therapy for Prolonging Allograft Survival 298
24 Fetal Gene Therapy 299
Introduction 299
Gene Transfer Into Fetal Organs 299
Table of Contents xiii
Hematopoietic Stem Cells (HSC) for Fetal Gene Therapy 300
Timing of Fetal Gene Therapy 300
Animal Models of Fetal Gene Therapy 300
Potential Human Applications 300
Fetal Gene Therapy for Cystic Fibrosis 301
Fetal Intestinal Gene Therapy 301
25 Gene Therapy for Muscular Dystrophies 303
Introduction 303
Cell Transplant for Duchenne Muscular Dystrophy 304
Gene Therapy Approaches to Duchenne Muscular Dystrophy 304
Routes of Administration 308
Future Directions 309
26 Disorders of Special Senses 311
Retinal Disorders 311
Methods of Gene Transfer to Retinal Cells 311
Retinitis Pigmentosa (RP) 311
Gene Therapy for Retinal Degeneration 312
Potential of Gene Therapy for Hearing Disorders 312
27 Ethical, Legal, and Regulatory Aspects of Gene Therapy 315
Ethical Aspects of Somatic Cell Gene Therapy 315
Ethical Aspects of Germline Gene Therapy 316
Legal Regulation of Gene Therapy 316
Regulation of Gene Therapy in the United States by the FDA 317
Regulation of Gene Therapy in Europe 317
Regulatory Issues for Genetically Engineered Cell Transplants 318
Safety Issues 318
Quality Control 321
Gene Therapy Patents 323
28 Research and Development in Gene Therapy 325
Introduction 325
Clinical Trials 325
Financing Research and Development 326
An Overview of the Development of Gene Therapy 326
International Development of Gene Therapy 327
Future of Gene Therapy 328
29 Companies Involved in Gene Therapy 331
Introduction 331
Selected Companies Involved in Gene Therapy 331
Collaborations of Companies Involved in Gene Therapy 347
Appendix I Protocols for Gene Therapy 351
Appendix II Addendum to Points to Consider in Human Somatic Cell and
Gene Therapy 371
Appendix III Sources of Information on Gene Therapy 387
References 391
Index 429
|
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| spelling | Jain, Kewal K. 1937- Verfasser (DE-588)114376093 aut Textbook of gene therapy K. K. Jain Seattle [u.a.] Hogrefe & Huber 1998 XVIII, 434 S. Ill. txt rdacontent n rdamedia nc rdacarrier Gene Therapy Gene therapy Gentherapie (DE-588)4296957-8 gnd rswk-swf (DE-588)4151278-9 Einführung gnd-content Gentherapie (DE-588)4296957-8 s DE-604 HBZ Datenaustausch application/pdf http://bvbr.bib-bvb.de:8991/F?func=service&doc_library=BVB01&local_base=BVB01&doc_number=007849403&sequence=000002&line_number=0001&func_code=DB_RECORDS&service_type=MEDIA Inhaltsverzeichnis |
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| title_full_unstemmed | Textbook of gene therapy K. K. Jain |
| title_short | Textbook of gene therapy |
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| topic | Gene Therapy Gene therapy Gentherapie (DE-588)4296957-8 gnd |
| topic_facet | Gene Therapy Gene therapy Gentherapie Einführung |
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