Contents

Foreword: The Past Recalls the Future v Phillip David Noguchi, M.D. Preface vii Contributors xiii

Part I. Delivery Systems and Therapeutic Strategies

1. Retroviral Vectors for Gene Therapy 1 Paula M. Cannon and W. French Anderson

2. Adenovirus Vectors for Gene Therapy 17 Neil R. Hackett and Ronald G. Crystal

3. Modified Adenoviruses for Gene Therapy 43

Akseli Hemminki, Joel N. Glasgow, and David T. Curiel

4. Helper-dependent Adenoviral Vectors for Gene Therapy 53 Philip Ng and Frank L. Graham

5. Adeno-associated Virus and AAV Vectors for Gene Delivery 71 Barrie J. Carter, Haim Burstein, and Richard W. Peluso

6. Engineering Herpes Simplex Viral Vectors for Therapeutic Gene Transfer 103 D. Wolfe, W. F. Goins, D. J. Fink, E. A. Burton, D. M. Krisky, and J. C. Glorioso

7. Lentiviral Vectors 131 Douglas Jolly

8. Ex Vivo Gene Therapy Using Myoblasts and Regulatable Retroviral Vectors 147 Clare R. Ozawa, Matthew L. Springer, and Helen M. Blau

9. Vaccinia Viral Vectors 165

J. Andrea McCart and David L. Bartlett

10. Baculovirus-mediated Gene Transfer: An Evolving New Concept 181

Kari J. Airenne, Anssi J. Mahonen, Olli H. Laitinen, and Seppo Yla-Herttuala

11. Bacteria as Vectors for Gene Therapy of Cancer 199 Annette Paschen, Dirk Schadendorf, and Siegfried Weiss

12. Polyfunctional Vectors for Gene Delivery 211 Barbara Demeneix and Jean-Paul Behr

13. Receptor-Targeted Polyplexes 223 Kurosh Ameri and Ernst Wagner

14. Electrokinetic Enhancement of Plasmid Delivery In Vivo 245 Ruxandra Draghia-Akli and Louis C. Smith

15. Gene Gun Technologies: Applications for Gene Therapy and Genetic Immunization 263 Michael A. Barry, Rana A. K. Singh, and Helen A. Andersson

16. Optimization of Nonviral Gene Therapeutics 287

Nancy Smyth Templeton

17. Molecular Interactions in Lipids, DNA, and DNA-Lipid Complexes 301 Rudolf Podgornik, D. Harries, H. H. Strey, and V. A. Parsegian

Part II. Other Therapeutic Strategies

18. Gene Therapy Applications of Ribozymes 333 Bruce A. Sullenger, Lynn Milich, and J. P. Jones III

19. Antisense Oligonucleotide-based Therapeutics 347

C. Frank Bennett, Eric Swayze, Richard Geary, Art A. Levin, Rahul Mehta, Ching-Leou Teng, Lloyd Tillman, and Greg Hardee

20. Suicide Gene Therapy 375 Bert W. O'Malley, Jr.

21. Selectable Markers for Gene Therapy 391

Michael M. Gottesman, Thomas Licht, Chava Kimchi-Sarfaty, Yi Zhou, Caroline Lee, Tzipora Shoshani, Peter Hafkemeyer, Christine A. Hrycyna, and Ira Pastan

Part III. Gene Expression and Detection

22. Ligand-dependent Transcription Switches and Their Potential for Gene Therapy 413 Carlo Toniatti, Fabio Palombo, Riccardo Cortese, and Gennaro Ciliberto

23. Monitoring Gene Therapy in Living Subjects with Molecular Imaging Technologies 447 S. Biswal and S. S. Gambhir

Part IV. Disease Targets and Therapeutic Strategies

24. Gene Transfer into Hematopoietic Stem Cells 481 David M. Bodine

25. Gene Therapy for Hematopoietic Disorders 501 Mitchell E. Horwitz and Harry L. Malech

26. Gene Therapy for Cardiovascular Disease and Vascular Grafts 515 Abeel A. Mangi and Victor J. Dzau

27. Gene Therapy for Cancer 531 Karsten Brand

28. Overcoming Barriers to Efficient Airway Epithelial Gene Transfer 585 Larry G. Johnson

29. Gene Therapy for Neurological Diseases 601 William J. Bowers and Howard J. Federoff

30. Principles of Gene Therapy for Inborn Errors of Metabolism 629 Jon A. Wolff and Cary O. Harding

31. Gene Therapy and HIV-1 Infection 641

Muhammad Mukhtar, Ralph Dornburg, and Roger J. Pomerantz

32. Cutaneous Gene Therapy for Skin and Systemic Disorders 657 William F. Buitrago and Dennis R. Roop

33. Gene Therapy for Childhood Onset Blindness 675 Jean Bennett

34. DNA Vaccines 681

J. Joseph Kim, Andrew Y. Choo, and David B. Weiner

Part V. Gene Therapy Clinical Trials and Regulatory Issues

35. Cardiovascular Gene Therapy 707

Pinak B. Shah, Kapildeo Lotun, and Douglas W. Losordo

36. Selectively Replicating Adenoviruses: Clinical Experience with Onyx015 and Future Directions 721 Leonard E. Post

37. Gene Therapy for the Treatment of Cancer: From Laboratory to Bedside 727 Raphael F. Rousseau, Ettore Biagi, Martin Pule, and Malcolm K. Brenner

38. Gene Therapy Clinical Trials for Cancer: Replacement of Tumor Suppressor Gene p53 743 Jack A. Roth, Susan F. Grammer, James A. Merritt, and Stephen G. Swisher

39. Ex Vivo Gene Therapy for Alzheimer's Disease 753 Peter Nakaji and Hoi Sang U

40. Prospects for Clinical Application of Synthetic Gene Delivery Vectors 761 Nelson S. Yew and Seng H. Cheng

41. Regulatory Aspects of Gene Therapy 773 Stephanie L. Simek and Maritza C. McIntyre

Part VI. Other Cell-based Therapies and Trials

42. Immunoisolated Cells as Gene Therapy for CNS Diseases 787 Dwaine F. Emerich

43. Hematopoietic Progenitor Cells 813

Shannon L. McKinney-Freeman, Steven B. Bradfute, and Margaret A. Goodell

44. Gene Marking Studies of Hematopoietic Cells 831 Catherine M. Bollard, Malcolm K. Brenner, and Helen E. Heslop

45. Gene-based Vaccination Against Malignant Melanoma 843 Dirk Schadendorf

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