Gene therapy for diseases of the lung:
This up-to-the-minute and comprehensive resource lucidly covers gene therapy for lung diseases from existing technologies delivering foreign DNA to the lungs via the airways or circulation to promising new approaches for the further development of safe and efficient gene delivery systems
Gespeichert in:
| Format: | Buch |
|---|---|
| Sprache: | English |
| Veröffentlicht: |
New York [u.a.]
Dekker
1997
|
| Schriftenreihe: | Lung biology in health and disease
104 |
| Schlagworte: | |
| Online-Zugang: | Inhaltsverzeichnis |
| Zusammenfassung: | This up-to-the-minute and comprehensive resource lucidly covers gene therapy for lung diseases from existing technologies delivering foreign DNA to the lungs via the airways or circulation to promising new approaches for the further development of safe and efficient gene delivery systems Containing over 1350 bibliographic citations, tables, drawings, and photographs, Gene Therapy for Diseases of the Lung is an indispensable volume for pulmonologists, geneticists, molecular and cell biologists, oncologists, internists, physiologists, scientists involved in gene therapy, and graduate and medical school students in these disciplines |
| Beschreibung: | XVIII, 427 S. Ill., graph. Darst. |
| ISBN: | 0824700600 |
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| 245 | 1 | 0 | |a Gene therapy for diseases of the lung |c ed. by Kenneth L. Brigham |
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| 490 | 1 | |a Lung biology in health and disease |v 104 | |
| 520 | 3 | |a This up-to-the-minute and comprehensive resource lucidly covers gene therapy for lung diseases from existing technologies delivering foreign DNA to the lungs via the airways or circulation to promising new approaches for the further development of safe and efficient gene delivery systems | |
| 520 | |a Containing over 1350 bibliographic citations, tables, drawings, and photographs, Gene Therapy for Diseases of the Lung is an indispensable volume for pulmonologists, geneticists, molecular and cell biologists, oncologists, internists, physiologists, scientists involved in gene therapy, and graduate and medical school students in these disciplines | ||
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Datensatz im Suchindex
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|---|---|
| adam_text | CONTENTS
Introduction Claude Lenfant Hi
Preface v
Contributors ix
Part One Overview
1. Human Gene Therapy: The Initial Concepts 3
W. French Anderson
I. Introduction 3
II. The 1960s 3
III. The 1970s 8
Bibliography 14
2. Discovering Genes That Cause Disease 17
Francis S. Collins
References 25
xiv Contents
Part Two Delivering Genes to Human Lungs
3. Adenoviral Vectors for Gene Therapy of Inherited and
Acquired Disorders of the Lung 29
David T. Curiel and Robert I. Garver, Jr.
I. Overview of Adenovirus Biology 29
II. Conventional Recombinant Adenovirus Vectors 30
III. Adenoviral Vectors for Gene Delivery to the Lung 32
IV. Gene Therapy Approaches Based on Adenovirus
Vectors for Inherited Pulmonary Disorders 35
V. Potential Utility of Adenoviral Vectors for Treatment
of Acquired Lung Conditions 39
VI. Strategies to Improve Adenoviral Vectors from Lung
Disorder Gene Therapy Approaches 41
References 46
4. Adeno Associated Virus Based Delivery Systems 53
Pamela L. Zeitlin
I. Introduction 53
II. AAV Structure 54
III. AAV Replication In Vivo 58
IV. Recombinant AAV Vectors 61
V. AAV Vector Expression in Brain 68
VI. AAV Vectors in Skeletal Muscle 68
VII. Cystic Fibrosis 68
VIII. AAV Vectors in Cancer 70
IX. Summary and Future Directions 70
References 71
5. Cellular Responses to Adenovirus Entry 83
Susanna Chiocca and Matthew Gotten
I. Introduction 83
II. The Virus Entry Mechanism 84
III. The Virus Is Internalized and Endosomal Escape
Must Occur 88
IV. The Role of a Low pH Step in Adenovirus Entry
Is Not Yet Clear 89
V. Cytoplasmic Transit 89
VI. Virus Uncoating and Nuclear Entry: The Role of
the Adenovirus Protease 89
Contents xv
VII. How These Changes Can Influence the Health of
the Cell 90
VIII. Efforts to Modulate Immune Responses to
Adenovirus Vectors, Survival of Modified Cells,
Avoiding the Immune System 91
IX. Conclusions, Future Directions 92
References 92
6. Cationic Lipid Based Gene Delivery: An Update 99
Xiang Gao
I. Introduction 99
II. New Cationic Lipids and Formulations 100
III. New DNA/Lipid Formulations 105
IV. DNA/Lipid/Polymer Formulations 105
V. Recent Progress on Mechanisms of Lipid Mediated
Transfection 107
VI. Problems and Future Development 109
References 110
7. Liposome/Viral Hybrid Gene Delivery Systems 113
ArleneA. Stecenko
I. Introduction 113
II. Cell Targeting and Entry 114
III. Endosomolysis 120
IV. Retention/Replication of Foreign DNA 124
References 129
Part Three Gene Therapy in the Lung: The Next Generation
8. Manipulating the Intracellular Trafficking of Nucleic Acids 135
Kathleen E. B. Meyer, Lisa S. Uyechi, and Francis C. Szoka, Jr.
I. Introduction 135
II. Review of the Cytoplasm 136
III. Transport of Oligodeoxynucleotides 139
IV. Nucleocytoplasmic Transport of Plasmid DNA 140
V. Expression of DNA in the Cytoplasm as a Means to
Circumvent Low Nuclear Uptake 143
VI. The Mechanics of Movement in the Cytoplasm 143
VII. Review of the Nucleus 146
VIII. Studying Nucleocytoplasmic Transport 152
xvi Contents
IX. Nuclear Transport of Viruses 160
X. Assembly of the Nuclear Envelope 163
XI. Fusion with the Nuclear Membrane 166
XII. Conclusion 168
References 168
9. Delivery of Genes Through the Lung Circulation 181
David M. Rodman and Elizabeth G. Nabel
I. Why Deliver Genes Via the Vasculature? 181
II. Endothelial Cell Implantation 182
III. Systemic Injection of Lipid/DNA Complexes 183
IV. Directed Delivery to the Lung Circulation 186
V. Summary 188
References 190
10. Targeting Gene Expression to the Lung 193
Jeffrey A. Whitsett and Stephan W. Glasser
I. Introduction 193
II. Cellular Targets in the Lung 194
III. Transcriptional Control of Gene Expression 196
IV. Proximal SP B Enhancer 202
V. Posttranscriptional Control of Gene Expression 204
VI. Summary 205
References 205
11. Strategies to Accomplish Targeted Gene Delivery
Employing Tropism Modified Adenoviral Vectors 209
Joanne T. Douglas and David T. Curiel
I. Limitations of Adenoviral Vectors for Pulmonary
Gene Therapy Applications 209
II. Tropism Modified Viral Vectors Can Be Constructed
to Achieve Targeted, Cell Specific Gene Delivery 210
III. The Pathway of Adenoviral Entry Is Partially
Characterized 215
IV. Adenoviral Cellular Binding and Internalization Can
Be Uncoupled from Subsequent Steps in Infection 217
V. Toward a Tropism Modified Adenoviral Vector for
Cell Specific Gene Delivery 218
VI. Conclusion 230
References 231
Contents ^j
Part Four Disease Directed Gene Therapy
12. Toward Correction of the Genetic Defect in Cystic Fibrosis 239
Larry G. Johnson and Richard C. Boucher
I. Introduction 239
II. Clinical Overview 239
III. Molecular Pathophysiology 241
IV. Therapeutic Approaches to Gene Therapy 244
V. Conclusions 255
References 256
13. Correction of the Genetic Defect in AIpha 1 Antitrypsin
Deficiency by Somatic Gene Therapy 267
Randy C. Eisensmith and Savio L. C. Woo
I. Physiological Function of hAAT and Pathogenesis
of Pulmonary Emphysema 267
II. Molecular Genetics of hAAT Deficiency 268
III. Enzyme Replacement Therapy for hAAT Deficiency 269
IV. Somatic Gene Therapy for hAAT Deficiency 270
References 278
14. Gene Therapy for Chronic Inflammatory Diseases of
the Lungs 285
Angelo E. Canonico
I. Introduction 285
II. Alphai Antitrypsin 286
III. Gene Therapy for cdAT Deficiency 287
IV. Cystic Fibrosis 293
V. Other Chronic Inflammatory Lung Diseases
Potentially Amenable to Gene Therapy 301
VI. Conclusion 302
References 302
15. Gene Therapy for Acute Diseases of the Lungs 309
Kenneth L. Brigham
I. Introduction 309
II. Rationale for Gene Therapy for Nongenetic Diseases 310
III. Design of DNA Delivery Systems for Treatment of
Acute Diseases of the Lungs 310
xviii Contents
IV. Gene Therapy for Respiratory Viral Infections 314
V. Gene Therapy for Acute Lung Injury 316
VI. Is DNA Really Just Another Drug? 320
References 321
16. Gene Therapy for Lung Cancer 323
Choon Taek Lee and David P. Carbone
I. Introduction 323
II. Immunity Inducing Gene Therapy 324
III. Directly Cytotoxic Gene Therapy 332
IV. Conclusion 338
References 338
Part Five Epilogue
17. The Regulatory Process and Gene Therapy 349
Suzanne Epstein
I. Introduction 349
II. Manufacturing and Quality Control of Gene
Therapy Products 350
III. Preclinical Animal Studies 352
IV. Vector Modification 353
V. Clinical Trials 355
VI. Well Characterized Products 356
VII. Conclusion 356
References 357
18. Some Issues Affecting Progress Toward Human Gene
Therapy and Potential Application to the Lung 359
Theodore Friedmann
I. Introduction 359
II. Lessons from Recent Clinical Studies 360
III. Technical Needs 360
IV. Conclusion 363
References 364
Author Index 367
Subject Index 399
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| spelling | Gene therapy for diseases of the lung ed. by Kenneth L. Brigham New York [u.a.] Dekker 1997 XVIII, 427 S. Ill., graph. Darst. txt rdacontent n rdamedia nc rdacarrier Lung biology in health and disease 104 This up-to-the-minute and comprehensive resource lucidly covers gene therapy for lung diseases from existing technologies delivering foreign DNA to the lungs via the airways or circulation to promising new approaches for the further development of safe and efficient gene delivery systems Containing over 1350 bibliographic citations, tables, drawings, and photographs, Gene Therapy for Diseases of the Lung is an indispensable volume for pulmonologists, geneticists, molecular and cell biologists, oncologists, internists, physiologists, scientists involved in gene therapy, and graduate and medical school students in these disciplines Gentherapie gtt Longziekten gtt Gene Therapy Lung Diseases therapy Lungs Diseases Gene therapy Lungenkrankheit (DE-588)4036660-1 gnd rswk-swf Gentherapie (DE-588)4296957-8 gnd rswk-swf (DE-588)4143413-4 Aufsatzsammlung gnd-content Lungenkrankheit (DE-588)4036660-1 s Gentherapie (DE-588)4296957-8 s DE-604 Brigham, Kenneth L. Sonstige oth Lung biology in health and disease 104 (DE-604)BV000008326 104 HBZ Datenaustausch application/pdf http://bvbr.bib-bvb.de:8991/F?func=service&doc_library=BVB01&local_base=BVB01&doc_number=007643853&sequence=000002&line_number=0001&func_code=DB_RECORDS&service_type=MEDIA Inhaltsverzeichnis |
| spellingShingle | Gene therapy for diseases of the lung Lung biology in health and disease Gentherapie gtt Longziekten gtt Gene Therapy Lung Diseases therapy Lungs Diseases Gene therapy Lungenkrankheit (DE-588)4036660-1 gnd Gentherapie (DE-588)4296957-8 gnd |
| subject_GND | (DE-588)4036660-1 (DE-588)4296957-8 (DE-588)4143413-4 |
| title | Gene therapy for diseases of the lung |
| title_auth | Gene therapy for diseases of the lung |
| title_exact_search | Gene therapy for diseases of the lung |
| title_full | Gene therapy for diseases of the lung ed. by Kenneth L. Brigham |
| title_fullStr | Gene therapy for diseases of the lung ed. by Kenneth L. Brigham |
| title_full_unstemmed | Gene therapy for diseases of the lung ed. by Kenneth L. Brigham |
| title_short | Gene therapy for diseases of the lung |
| title_sort | gene therapy for diseases of the lung |
| topic | Gentherapie gtt Longziekten gtt Gene Therapy Lung Diseases therapy Lungs Diseases Gene therapy Lungenkrankheit (DE-588)4036660-1 gnd Gentherapie (DE-588)4296957-8 gnd |
| topic_facet | Gentherapie Longziekten Gene Therapy Lung Diseases therapy Lungs Diseases Gene therapy Lungenkrankheit Aufsatzsammlung |
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