Gene Therapy For Diseases Of The Lung: Brigham, Kenneth: 9780824700607: Book: Medical: eCampus.com

Vanderbilt Univ., Nashville, TN. Comprehensive text on gene therapy for lung diseases. Covers established and new technologies.

Introduction

Claude Lenfant

iii

Preface

Contributors

Part One Overview

1. Human Gene Therapy: The Initial Concepts

(14)

W. French Anderson

I. Introduction

(1)

II. The 1960's

(5)

III. The 1970's

(6)

Bibliography

(3)

2. Discovering Genes That Cause Disease

(12)

Francis S. Collins

References

(4)

Part Two Delivering Genes to Human Lungs

3. Adenoviral Vectors for Gene Therapy of Inherited and Acquired Disorders of the Lung

(24)

David T. Cunel and Roben I. Garver, Jr.

I. Overview of Adenovirus Biology

(1)

II. Conventional Recombinant Adenovirus Vectors

(2)

III. Adenoviral Vectors for Gene Delivery to the Lung

(3)

IV. Gene Therapy Approaches Based on Adenovirus Vectors for Inherited Pulmonary Disorders

(4)

V. Potential Utility of Adenoviral Vectors for Treatment of Acquired Lung Conditions

(2)

VI. Strategies to Improve Adenoviral Vectors from Lung Disorder Gene Therapy Approaches

(5)

References

(7)

4. Adeno-Associated Virus-Based Delivery Systems

(30)

Pamela L. Zeitlin

I. Introduction

(1)

II. AAV Structure

(4)

III. AAV Replication In Vivo

(3)

IV. Recombinant AAV Vectors

(7)

V. AAV Vector Expression in Brain

(1)

VI. AAV Vectors in Skeletal Muscle

(1)

VII. Cystic Fibrosis

(2)

VIII. AAV Vectors in Cancer

(1)

IX. Summary and Future Directions

(1)

References

(12)

5. Cellular Responses to Adenovirus Entry

(16)

Susanna Chiocca and Matthew Gotten

I. Introduction

(1)

II. The Virus Entry Mechanism

(4)

III. The Virus Is Internalized and Endosomal Escape Must Occur

(1)

IV. The Role of a Low pH Step in Adenovirus Entry Is Not Yet Clear

(1)

V. Cytoplasmic Transit

(1)

VI. Virus Uncoating and Nuclear Entry: The Role of the Adenovirus Protease

(1)

VII. How These Changes Can Influence the Health of the Cell

(1)

VIII. Efforts to Modulate Immune Responses to Adenovirus Vectors, Survival of Modified Cells, Avoiding the Immune System

(1)

IX. Conclusions, Future Directions

(1)

References

(7)

6. Cationic Lipid-Based Gene Delivery: An Update

(14)

Xiang Gao

I. Introduction

(1)

II. New Cationic Lipids and Formulations

100

(5)

III. New DNAJLipid Formulations

105

(1)

IV. DNA/Lipid/Polymer Formulations

105

(2)

V. Recent Progress on Mechanisms of Lipid-Mediated Transfection

107

(2)

VI. Problems and Future Development

109

(1)

References

110

(3)

7. Liposome/Viral Hybrid Gene Delivery Systems

113

(22)

Arlene A. Stecenko

I. Introduction

113

(1)

II. Cell Targeting and Entry

114

(6)

III. Endosomolysis

120

(4)

IV. Retention/Replication of Foreign DNA

124

(5)

References

129

(6)

Part Three Gene Therapy in the Lung: The Next Generation

8. Manipulating the Intracellular Trafficking of Nucleic Acids

135

(46)

Kathleen E.B. Meyer, Lisa S. Uyechi, and Francis C. Szoka, Jr.

I. Introduction

135

(1)

II. Review of the Cytoplasm

136

(3)

III. Transport of Oligodeoxynucleotides

139

(1)

IV. Nucleocytoplasmic Transport of Plasmid DNA

140

(3)

V. Expression of DNA in the Cytoplasm as a Means to Circumvent Low Nuclear Uptake

143

(1)

VI. The Mechanics of Movement in the Cytoplasm

143

(3)

VII. Review of the Nucleus

146

(6)

VIII. Studying Nucleocytoplasmic Transport

152

(8)

IX. Nuclear Transport of Viruses

160

(3)

X. Assembly of the Nuclear Envelope

163

(3)

XI. Fusion with the Nuclear Membrane

166

(2)

XII. Conclusion

168

(1)

References

168

(13)

9. Delivery of Genes Through the Lung Circulation

181

(12)

David M. Rodman and Elizabeth G. Nabel

I. Why Deliver Genes Via the Vasculature?

181

(1)

II. Endothelial Cell Implantation

182

(1)

III. Systemic Injection of Lipid/DNA Complexes

183

(3)

IV. Directed Delivery to the Lung Circulation

186

(2)

V. Summary

188

(2)

References

190

(3)

10. Targeting Gene Expression to the Lung

193

(16)

Jeffrey A. Whitsett and Stephan W. Glasser

I. Introduction

193

(1)

II. Cellular Targets in the Lung

194

(2)

III. Transcriptional Control of Gene Expression

196

(6)

IV. Proximal SP-B Enhancer

202

(2)

V. Posttranscriptional Control of Gene Expression

204

(1)

VI. Summary

205

(1)

References

205

(4)

11. Strategies to Accomplish Targeted Gene Delivery Employing Tropism-Modified Adenoviral Vectors

209

(30)

Joanne T. Douglas and David T. Cunel

I. Limitations of Adenoviral Vectors for Pulmonary Gene Therapy Applications

209

(1)

II. Tropism-Modified Viral Vectors Can Be Constructed to Achieve Targeted, Cell-Specific Gene Delivery

210

(5)

III. The Pathway of Adenoviral Entry Is Partially Characterized

215

(2)

IV. Adenoviral Cellular Binding and Internalization Can Be Uncoupled from Subsequent Steps in Infection

217

(1)

V. Toward a Tropism-Modified Adenoviral Vector for Cell-Specific Gene Delivery

218

(12)

VI. Conclusion

230

(1)

References

231

(8)

Part Four Disease-Directed Gene Therapy

12. Toward Correction of the Genetic Defect in Cystic Fibrosis

239

(28)

Larry G. Johnson and Richard C. Boucher

I. Introduction

239

(1)

II. Clinical Overview

239

(2)

III. Molecular Pathophysiology

241

(3)

IV. Therapeutic Approaches to Gene Therapy

244

(11)

V. Conclusions

255

(1)

References

256

(11)

13. Correction of the Genetic Defect in Alpha-1 Antitrypsin Deficiency by Somatic Gene Therapy

267

(18)

Randy C. Eisensmith and Savio L.C. Woo

I. Physiological Function of hAAT and Pathogenesis of Pulmonary Emphysema

267

(1)

II. Molecular Genetics of hAAT Deficiency

268

(1)

III. Enzyme Replacement Therapy for hAAT Deficiency

269

(1)

IV. Somatic Gene Therapy for hAAT Deficiency

270

(8)

References

278

(7)

14. Gene Therapy for Chronic Inflammatory Diseases of the Lungs

285

(24)

Angelo E. Canonico

I. Introduction

285

(1)

II. Alphas Antitrypsin

286

(1)

III. Gene Therapy for a1AT Deficiency

287

(6)

IV. Cystic Fibrosis

293

(8)

V. Other Chronic Inflammatory Lung Diseases Potentially Amenable to Gene Therapy

301

(1)

VI. Conclusion

302

(1)

References

302

(7)

15. Gene Therapy for Acute Diseases of the Lungs

309

(14)

Kenneth L. Brigham

I. Introduction

309

(1)

II. Rationale for Gene Therapy for Nongenetic Diseases

310

(1)

III. Design of DNA Delivery Systems for Treatment of Acute Diseases of the Lungs

310

(4)

IV. Gene Therapy for Respiratory Viral Infections

314

(2)

V. Gene Therapy for Acute Lung Injury

316

(4)

VI. Is DNA Really Just Another Drug?

320

(1)

References

321

(2)

16. Gene Therapy for Lung Cancer

323

(26)

Choon-Taek Lee and David P. Carbone

I. Introduction

323

(1)

II. Immunity-Inducing Gene Therapy

324

(8)

III. Directly Cytotoxic Gene Therapy

332

(6)

IV. Conclusion

338

(1)

References

338

(11)

Part Five Epilogue

17. The Regulatory Process and Gene Therapy

349

(10)

Suzanne Epstein

I. Introduction

349

(1)

II. Manufacturing and Quality Control of Gene Therapy Products

350

(2)

III. Preclinical Animal Studies

352

(1)

IV. Vector Modification

353

(2)

V. Clinical Trials

355

(1)

VI. "Well-Characterized Products"

356

(1)

VII. Conclusion

356

(1)

References

357

(2)

18. Some Issues Affecting Progress Toward Human Gene Therapy and Potential Application to the Lung

359

(8)

Theodore Friedmann

I. Introduction

359

(1)

II. Lessons from Recent Clinical Studies

360

(1)

III. Technical Needs

360

(3)

IV. Conclusion

363

(1)

References

364

(3)

Author Index

367

(32)

Subject Index

399




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