Hematopoiesis and gene therapy:
Saved in:
| Format: | Book |
|---|---|
| Language: | English |
| Published: |
New York, N.Y. [u.a.]
Kluwer Acad., Plenum Publ.
1999
|
| Series: | Blood cell biochemistry
8 |
| Subjects: | |
| Online Access: | Inhaltsverzeichnis |
| Physical Description: | XVI, 380 S. Ill., graph. Darst. |
| ISBN: | 0306459620 |
Staff View
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| 245 | 1 | 0 | |a Hematopoiesis and gene therapy |c ed. by Leslie J. Fairbairn ... |
| 264 | 1 | |a New York, N.Y. [u.a.] |b Kluwer Acad., Plenum Publ. |c 1999 | |
| 300 | |a XVI, 380 S. |b Ill., graph. Darst. | ||
| 336 | |b txt |2 rdacontent | ||
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| 490 | 1 | |a Blood cell biochemistry |v 8 | |
| 650 | 4 | |a Gene Therapy | |
| 650 | 4 | |a Gene Transfer Techniques | |
| 650 | 4 | |a Gene therapy | |
| 650 | 4 | |a Hematopoiesis | |
| 650 | 4 | |a Hematopoietic Stem Cell Transplantation | |
| 650 | 4 | |a Immunotherapy | |
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Record in the Search Index
| _version_ | 1804127350761193472 |
|---|---|
| adam_text | Contents
Chapter 1
Hemopoietic Stem Cells as Targets for Genetic Manipulation:
Concepts and Practical Approaches
N. G. Testa, E. A. de Wynter, and J. Hows
1. How Many Stem Cells Do We Need? 1
1.1. The Experimental Approach 1
1.2. Patient Data 2
2. Selection and Identification by Phenotype and Function 3
3. Sources of Primitive Hematopoietic Cells 5
3.1. Bone Marrow 5
3.2. Peripheral Blood 5
3.3. Cord Blood 6
4. How Can We Maximize the Number of Target Cells? 6
5. Concluding Comments 9
6. References 9
Chapter 2
Bone Marrow Transplantation for Genetic Diseases
Colin G. Steward
1. Introduction 13
2. General Principles of BMT 14
2.1. Classes of Diseases Treated 14
2.2. Enzyme Transfer 14
2.3. Animal Models 15
2.4. Mechanism and Speed of Response 16
2.5. Displacement BMT 17
ix
x Contents
2.6. Conditioning Therapy 18
2.7. Studies of Graft Chimerism after BMT 19
2.8. Immunoprophylaxis 20
3. The Bone Marrow Transplantation Process 21
3.1. Patient Assessment 21
3.2. Donor Selection 22
3.3. Transplantation Protocol 23
4. Complications of BMT 25
4.1. Short and Medium Term Complications 25
4.2. Late Effects of BMT 28
5. Results of BMT 29
5.1. Disorders of Hematopoiesis 30
5.2. Disorders of Fixed Tissue Cells of
Monocyte/Macrophage Origin 38
5.3. Metabolic Disorders 41
6. Future Directions 46
6.1. Graft Manipulation 46
6.2. In Utero Transplantation 46
6.3. Cord Blood Transplantation 47
7. References 48
Chapter 3
Retroviral Vectors
Mary Collins and Colin Porter
1. Why Retroviruses? 57
2. The Basic Vector 58
2.1. Minimal Vector Requirements 58
2.2. Strategies for Vector Design 62
3. The Packaging Cell 67
3.1. Viral Proteins 68
3.2. Packaging Cell Lines 72
4. References 78
Chapter 4
Parvoviral Vectors for Human Hematopoietic Gene Therapy
Arun Srivastava
1. Introduction 89
2. Life Cycle of Human Parvoviruses 90
2.1. Infection and Host Cell Receptors 91
2.2. DNA Replication and Gene Expression 92
2.3. Viral Assembly 93
2.4. Proviral Integration, Rescue, and Replication 94
fci. i
Contents xi
3. Recombinant Parvoviral Vectors 94
3.1. Construction Strategies 94
3.2. Rescue and Packaging Cell Lines 95
3.3. High Titer Vector Stocks and Viral Purification 95
3.4. Defective Interfering Particles 96
4. Parvovirus Mediated Transduction and Expression of Genes 97
4.1. Nature of the Proviral Genome 98
4.2. Site Specificity of Integration 98
4.3. Tissue Specificity of Gene Expression 100
4.4. Gene Therapy of Human Hemoglobinopathies 103
5. Advantages and Disadvantages of Parvovirus Vectors Ill
5.1. Size Limitation Ill
5.2. Large Scale Production 112
5.3. Host Range 112
5.4. Safety and Efficacy 112
6. Summary and Conclusions 112
7. Future Prospects 113
8. References 113
Chapter 5
Nonviral Methods for Gene Transfer
A. Djeha and L. S. Lashford
1. Introduction 123
2. Plasmid DNA Based Methods 124
2.1. Physical Methods 124
2.2. Direct Injection of Naked DNA 126
3. Artificial Self Assembling Systems 129
3.1. Lipid Mediated Gene Delivery Methods 129
3.2. Ligand Mediated Gene Delivery Methods 135
4. References 142
Chapter 6
Prospects for Gene Therapy of Inherited Immunodeficiency
Colin Casimir
1. Introduction to Gene Therapy 155
1.1. Immunodeficiencies as Candidate Disorders for
Gene Therapy 155
1.2. Target Cell Populations 156
2. Immunodeficiency Disorders 157
2.1. Severe Combined Immunodeficiency (SCID) 157
2.2. Defects of Purine Metabolism 159
2.3. B and T Cell Immunodeficiencies 161
xjj Contents
2.4. Myeloid Cell Specific Disorders 165
3. Vector Systems and Their Suitability for Gene
Therapy of Immunodeficiency 170
4. Gene Therapy for Immunodeficiency Disorders 171
4.1. ADA SCID 171
4.2. Gene Therapy for Non ADA Diseases 174
4.3. Retrovirally Mediated Gene Transfer into
Hematopoietic Stem Cells in Vitro 175
4.4. Clinical Gene Marking Trials 177
4.5. Strategies to Overcome the Low Level of Gene
Transfer to Human PHSC 178
5. Conclusion 181
6. References 182
Chapter 7
Gene Therapy for Lysosomal Disorders
L. J. Fairbairn and L. S. Lashford
1. Defining a Population of Patients Suitable for Treatment 203
1.1. The Relationship between Genotype and
Expected Clinical Outcome 204
1.2. The Availability of Alternative Treatments 204
2. Gene Therapy 208
2.1. Autologous Hematopoietic Stem Cells 208
2.2. Lymphocyte Gene Transfer 210
2.3. Neo Organs 211
3. Targeting the Brain 211
3.1. Bone Marrow Macrophages 211
3.2. Neurotropic Vectors 212
3.3. Neural Progenitor Cells 213
4.. Conclusions 214
5. References 214
Chapter 8
Genetic Approaches to Therapy for the Hemoglobinopathies
Michael Antoniou and Frank Grosveld
1. Introduction 219
2. Current Therapies for P Thalassaemia 220
2.1. Blood Transfusions and Iron Chelation 220
2.2. Allogeneic Hematopoietic Stem Cell Transplantation 220
3. Current Therapies for Sickle Cell Disease 222
3.1. Antisickling Agents 222 i
4. Regulation of Gene Expression from the Human
p Globin Locus 222 I
i
Contents xiii
5. Pharmaceutical Activation of Fetal y Globin Gene
Expression in the Adult 226
6. Gene Therapy 228
7. Summary and Future Prospects 232
8. References 234
Chapter 9
Gene Marking and the Biology of Hematopoietic Cell Transfer in
Human Clinical Trials
A. K. Stewart, I. D. Dube, and R. G. Hawley
1. Introduction 243
2. Gene Marking and Cancer Biology , 244
3. Retroviral Marking of Tumor Infiltrating Lymphocytes 244
4. Clinical Trials of Stem Cell Gene Marking 245
5. Clinical Trial Design 246
6. Retroviral Marking of Malignant Cells 248
7. Clinical Trial Results 249
8. Hematopoiesis and Gene Marking 249
9. Genetically Marked Relapse 250
10. Retroviral Gene Marking and Purging Efficacy 252
11. Implications of Gene Marking Results in Clinical Practice 253
12. In Vitro Strategies to Improve Hematopoietic
Stem Cell Targeting 253
12.1. In Vitro Cycling 253
12.2. In Vivo Cycling 254
12.3. Stromal Layers or Fibronectin 255
12.4. Stem Cell Isolation 255
12.5. Cell to Retrovirus Contact 256
12.6. Pseudotyped Recombinant Retroviruses 256
12.7. MSCV Retroviral Vector System 257
12.8. Targeting with Engineered Retroviral Envelope Proteins 257
12.9. Alternative Vector Systems 258
13. Future Directions 258
14. Summary 259
15. References 259
Chapter 10
Antisense Strategies to Leukemia
Stephen G. O Brien
1. Introduction 269
2. Antisense and Ribozymal Design and Mechanisms of Action 270
2.1. Oligonucleotide Analogs and Stability 271
2.2. Cellular Uptake 272
2.3. Mechanisms of Target Inactivation
xjv Contents
by Antisense Oligomers 272
2.4. Ribozymal Principles and Design 273
3. Controls, Experimental Systems, and Difficulties
in Interpretation 274
3.1. Controls 274
3.2. Test Systems 275
3.3. Difficulties in Interpretation 275
4. Antisense and Ribozymes in Experimental Hematology 277
5. Clinical Trials in Hematology 280
6. Future Prospects 282
7. References 285
Chapter 11
Transfer of Drug Resistance Genes into Bone Marrow Stem and
Progenitor Cells: Implications for Cancer Chemotherapy
J. A. Rafferty and L. J. Fairbairn
1. Introduction 293
2. Current Strategies for Hematopoietic
Support during Chemotherapy 294
2.1. Blood and Associated Products 294
2.2. Peptide Factors 295
2.3. Bone Marrow and Peripheral Blood Progenitor
Cell Transplantation 296
2.4. Perspective 296
3. Gene Transfer, Expression, and Stem Cell Protection 297
3.1. Gene Transfer into Hematopoietic Cells 297
3.2. Genes Conferring Drug Resistance 298
4. Further Considerations 305
4.1. General Advantages 305
4.2. The Next Dose Limiting Toxicities 305
4.3. Target Cell Selectability 306
4.4. Therapy Related Malignancy 307
5. Concluding Remarks 307
6. References 307
Chapter 12
HIV Gene Therapy Using Hairpin Ribozymes in Hematopoietic
Stem/Progenitor Cells
Xinqiang Li, Flossie Wong Staal, Anthony D. Ho, and Ping Law
1. Ribozyme for Gene Therapy of AIDS 313
1.1. Ribozymes 313
1.2. Factors Affecting Ribozymal Gene Therapy 314
i
|
fc J
1
Contents xv
2. Gene Therapy Using Hematopoietic Stem/Progenitor Cells 315
2.1. Identification of Stem/Progenitor Cells 316
2.2. Enrichment of Stem/Progenitor Cells 316
2.3. Stimulation of Stem/Progenitor Cells for Transduction 317
2.4. Preparation of Vectors and Hematopoietic Stem Cells 318
2.5. Expression in Progeny Cells 319
2.6. Clinical Gene Marking/Gene Therapy Trials 319
3. Transduction of Stem/Progenitor Cells Using
Ribozymal Constructs 320
4. Toward Clinical Trials 321
4.1. HIV Infection of Stem/Progenitor Cells 321
4.2. Mobilization of Peripheral Blood Stem
Cells from AIDS Patients 322
4.3. Clinical Trials and Further Developments 322
5. References 323
Chapter 13
Molecular Immunotherapy by Gene Transfer
Rosa Maria Diaz and Richard G. Vile
1. Introduction 331
2. Are Tumors Antigenic? 332
3. Antigen Presentation Pathways 335
4. Immunotherapy by Gene Transfer 336
4.1. Gene Transfer of Components of the Antigen Presentation
Apparatus 340
4.2. Cytokines 340
4.3. Gene Transfer of Nonspecific Immunogens 341
4.4. Direct Cell Killing 341
4.5. Gene Transfer of Cloned Tumor Antigens 342
5. Aims, Goals, and Targets for Gene Transfer in
Immunotherapy 343
6. Unification of Diverse Results from Gene
Transfer Studies 344
7. References 345
Chapter 14
DNA Based Immunization
Heather L. Davis and Cynthia L. Brazolot Millan
1. Introduction 351
1.1. What is DNA Based Immunization? 351
1.2. Goals of Immunization 351
xvi Contents
1.3. Shortcomings of Antigen Based Vaccines 353
1.4. Unique Features of DNA Based Immunization 355
1.5. Potential Applications for DNA Vaccines 356
1.6. History of DNA Vaccines 356
1.7. Gene Transfer Methods for DNA Based Immunization 357
1.8. Design of Plasmid Vectors for DNA Based
Immunization 358
2. DNA Vaccines for Protection against Infectious
Diseases 359
3. DNA Vaccines for Immunotherapy 365
3.1. Treatment of Chronic Viral Infections 365
3.2. Immunotherapy for Cancer 367
4. Mechanism of Induction of Immune Responses 367
4.1. Antigen Presentation and T Cell Responses 367
4.2. B Cell Responses with Secreted and
Nonsecreted Antigens 368
5. Safety Considerations 368
5.1. Possibility of Tolerance to Foreign Antigen 368
5.2. Possibility of an Integrative Event 368
5.3. Possibility of Immune Response to DNA and
Autoimmunity 369
5.4. Other Possible Responses to Injected DNA 369
6. Summary and Future Directions 370
7. Implications for Gene Therapy 371
8. References 372
Index 377
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| indexdate | 2024-07-09T18:31:57Z |
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| isbn | 0306459620 |
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| physical | XVI, 380 S. Ill., graph. Darst. |
| publishDate | 1999 |
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| series | Blood cell biochemistry |
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| spelling | Hematopoiesis and gene therapy ed. by Leslie J. Fairbairn ... New York, N.Y. [u.a.] Kluwer Acad., Plenum Publ. 1999 XVI, 380 S. Ill., graph. Darst. txt rdacontent n rdamedia nc rdacarrier Blood cell biochemistry 8 Gene Therapy Gene Transfer Techniques Gene therapy Hematopoiesis Hematopoietic Stem Cell Transplantation Immunotherapy Blutbildendes Gewebe (DE-588)4239650-5 gnd rswk-swf Gentherapie (DE-588)4296957-8 gnd rswk-swf Blutbildendes Gewebe (DE-588)4239650-5 s Gentherapie (DE-588)4296957-8 s DE-604 Fairbairn, Leslie J. Sonstige oth Blood cell biochemistry 8 (DE-604)BV004308853 8 HBZ Datenaustausch application/pdf http://bvbr.bib-bvb.de:8991/F?func=service&doc_library=BVB01&local_base=BVB01&doc_number=008624432&sequence=000002&line_number=0001&func_code=DB_RECORDS&service_type=MEDIA Inhaltsverzeichnis |
| spellingShingle | Hematopoiesis and gene therapy Blood cell biochemistry Gene Therapy Gene Transfer Techniques Gene therapy Hematopoiesis Hematopoietic Stem Cell Transplantation Immunotherapy Blutbildendes Gewebe (DE-588)4239650-5 gnd Gentherapie (DE-588)4296957-8 gnd |
| subject_GND | (DE-588)4239650-5 (DE-588)4296957-8 |
| title | Hematopoiesis and gene therapy |
| title_auth | Hematopoiesis and gene therapy |
| title_exact_search | Hematopoiesis and gene therapy |
| title_full | Hematopoiesis and gene therapy ed. by Leslie J. Fairbairn ... |
| title_fullStr | Hematopoiesis and gene therapy ed. by Leslie J. Fairbairn ... |
| title_full_unstemmed | Hematopoiesis and gene therapy ed. by Leslie J. Fairbairn ... |
| title_short | Hematopoiesis and gene therapy |
| title_sort | hematopoiesis and gene therapy |
| topic | Gene Therapy Gene Transfer Techniques Gene therapy Hematopoiesis Hematopoietic Stem Cell Transplantation Immunotherapy Blutbildendes Gewebe (DE-588)4239650-5 gnd Gentherapie (DE-588)4296957-8 gnd |
| topic_facet | Gene Therapy Gene Transfer Techniques Gene therapy Hematopoiesis Hematopoietic Stem Cell Transplantation Immunotherapy Blutbildendes Gewebe Gentherapie |
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