| Foreword: Genetic Therapy in the 21st Century |
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iii | (2) |
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| Preface: From Theory to Practice in Thirty Years |
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v | (8) |
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| Contributors |
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xiii | |
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1. A Brief History of Genetic Therapy: Gene Therapy, Antisense Technology, and Genomics |
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1 | (38) |
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2. Preclinical Development of Antisense Oligonucleotide Therapeutics for Cancer: Regulatory Aspects |
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39 | (14) |
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3. Commercial Scale Manufacturing of Oligonucleotides Under Good Manufacturing Practices |
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53 | (36) |
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4. The Regulatory Process and Gene Therapy |
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89 | (12) |
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5. Production of Clinical Lots of Gene Therapy Vectors Using Good Manufacturing Practice: Experience in a University Setting |
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101 | (10) |
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6. Gene Therapy Clinical Trials for Adenosine Deaminase Deficiency/Severe Combined Immunodeficiency |
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111 | (22) |
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7. Development of an Oligodeoxynucleotide Pharmaceutical for the Treatment of Human Leukemia |
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133 | (22) |
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8. Clinical Trials with Anti-p53 DNA, OL(1)p53, in Patients with Acute Myelogenous Leukemia and Myelodysplastic Syndrome |
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155 | (14) |
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9. Human Bcl-2 Antisense Therapy for Lymphomas |
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169 | (22) |
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10. Retroviral Gene Transfer in Autologous Bone Marrow and Stem Cell Transplantation |
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191 | (28) |
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11. Adenoviral Gene Transfer of the Herpes Virus Thymidine Kinase Gene for Treating Gliomas |
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219 | (20) |
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12. Distribution and Toxicity of Retroviral Vectors After Intracavitary Delivery in Mouse and Man |
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239 | (18) |
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13. Clinical and Immunologic Responses to Gene Transfer of an Allogeneic Major Histocompatibility Complex Antigen |
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257 | (22) |
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14. Defective Tumor Suppressor Gene Replacement and Oncogene Inactivation for the Treatment of Cancer |
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279 | (16) |
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15. The Molecular Basis of Bladder Cancer and Prospects for Gene Therapy Using Hammerhead Ribozymes |
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295 | (16) |
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16. In Situ Gene Insertion for Immunotherapy Using Vaccinia Virus Vectors |
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311 | (20) |
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17. Antisense Oligonucleotide-Based Therapy for HIV-1 Infection from Laboratory to Clinical Trials |
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331 | (22) |
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18. Treatment of Retinitis Induced by Cytomegalovirus Using Intravitreal Fomivirsen (Isis 2922) |
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353 | (10) |
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19. Synthetic DNA-Based Compounds for the Prevention of Coronary Restenosis: Current Status and Future Challenges |
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363 | (32) |
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20. Prevention of Restenosis by Gene Targeting |
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395 | (18) |
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| Index |
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413 | |