Clinical trials of genetic therapy with antisense DNA and DNA vectors

A Brief History of Genetic Therapy: Gene Therapy, Antisense Technology, and Genomics, James W. HawkinsPreclinical Development of Oligonucleotide Therapeutics for Cancer: Regulatory Aspects, Chang-Ho Ahn and Joseph J. DeGeorgeCommercial Scale Manufacturing of Oligonucleotides Under Good Manufacturing Practices, Jose E. Gonzalez, Richard G. Einig, Patricia Puma, Timothy P.Noonan, Paul E. Kennedy, Bruce G. Sturgeon, Bing H. Wang, and Jin-yan TangThe Regulatory Process and Gene Therapy, Suzanne L. EpsteinProduction of Clinical Lots of Gene Therapy Vectors Using Good Manufacturing Practice: Experience in a University Setting, Alan R. Davis and Colleen BakerGene Therapy Clinical Trials for Adenosine Deaminase Deficiency/Severe Combined Immunodeficiency, Erlinda M. Gordon and W. French AndersonDevelopment of an Oligodeoxynucleotide Pharmaceutical for the Treatment of Human Leukemia, Alan M. Gewirtz and Deborah Lee SokolClinical Trials with Anti-p53 DNA, OL(1)p53, in Patients with Acute Myelogenous Leukemia and Myelodysplastic Syndrome, Patrick L. IversenHuman Bcl-2 Antisense Therapy for Lymphomas, Finbarr E. Cotter, Andrew Webb, Paul Clarke, and David CunninghamRetroviral Gene Transfer in Autologous Bone Marrow and Stem Cell Transplantation, Rafat Abonour and Kenneth CornettaAdenoviral Gene Transfer of the Herpes Virus Thymidine Kinase Gene for Treating Gliomas, Jane B. Alavi, Jason G. Smith, and Stephen L. EckDistribution and Toxicity of Retroviral Vectors After Intracavitary Delivery in Mouse and Man, Patrice S. Obermiller, Anne M. Pilaro, Carlos L. Arteaga, and Jeffrey T. HoltClinical and Immunologic Responses to Gene Transfer of an Allogeneic Major Histocompatibility Complex Antigen, Alison T. Stopeck and Evan M. HershDefective Tumor Suppressor Gene Replacement and Oncogene Inactivation for the Treatment of Cancer, Jack RothThe Molecular Basis of Bladder Cancer and Prospects for Gene Therapy Using Hammerhead Ribozymes, Eric J. Small, Mohammed Kashani-Sabet, David Y. Bouffard, and Kevin J. ScanlonIn Situ Gene Insertion for Immunotherapy Using Vaccinia Virus Vectors, Edmund C. Lattime, Laurence C. Eisenlohr, and Michael J. MastrangeloAntisense Oligonucleotide-Based Therapy for HIV-1 Infection from Laboratory to Clinical Trials, Sudhir AgrawalTreatment of Retinitis Induced by Cytomegalovirus Using Intravitreal Fomivirsen (Isis 2922), Stanley T. CrookeSynthetic DNA-Based Compounds for the Prevention of Coronary Restenosis: Current Status and Future Challenges, Andrew Zalewski, Yi Shi, John D. Mannion, and Fernando RoquéPrevention of Restenosis by Gene Targeting, Michael J. Mann, Heiko E. Von der Leyen, and Victor J. Dzau;An important new collection of clinical and preclinical reports on genetic therapy, this book describes illustrative examples of diseases in which gene-based interventions are presently plausible, and presents case studies of current research using both synthetic oligonucleotides and biological vectors. Combining the insights of over 50 contributors, Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectorsfurnishes a historical overview of genetic therapy highlights official Food and Drug Administration positions on the preparation of oligonucleotides and vectors offers practical models of agent preparation, animal testing, pharmacokinetics, toxicology, and clinical trials discusses both synthetic DNA and biological vector approaches to cancer, viral, and cardiological indications illustrates for new practitioners how each stage of genetic therapy is developed details genetic treatment of leukemia; lymphoma; cancer of the brain, breast, colon, kidney, and lung; melanoma; HIV; and coronary restenosis includes examples of antisense, ribozyme, tumor suppressor, immunostimulation, and gene replacement therapy and addresses questions of preparation, delivery, toxicity, mechanism, and specificity. Cover......Page 1 Half Title......Page 2 Title Page......Page 4 Copyright Page......Page 5 Foreword: Genetic Therapy in the 21st Century......Page 6 Preface: From Theory to Practice in Thirty Years......Page 8 Contents......Page 12 Contributors......Page 16 1. A Brief History of Genetic Therapy: Gene Therapy, Anti-sense Technology, and Genomics......Page 22 2. Preclinical Development of Antisense Oligonucleotide Therapeutics for Cancer: Regulatory Aspects......Page 60 3. Commercial Scale Manufacturing of Oligonucleotides Under Good Manufacturing Practices......Page 74 4. The Regulatory Process and Gene Therapy......Page 110 5. Production of Clinical Lots of Gene Therapy Vectors Using Good Manufacturing Practice: Experience in a University Setting......Page 122 6. Gene Therapy Clinical Trials for Adenosine Deaminase Deficiency/Severe Combined Immunodeficiency......Page 132 7. Development of an Oligodeoxynucleotide Pharmaceutical for the Treatment of Human Leukemia......Page 154 8. Clinical Trials with Anti-p53 DNA, OL(1)p53, in Patients with Acute Myelogenous Leukemia and Myelodysplastic Syndrome......Page 176 9. Human Bcl-2 Antisense Therapy for Lymphomas......Page 190 10. Retroviral Gene Transfer in Autologous Bone Marrow and Stem Cell Transplantation......Page 212 11. Adenoviral Gene Transfer of the Herpes Virus Thymidine Kinase Gene for Treating Gliomas......Page 240 12. Distribution and Toxicity of Retroviral Vectors After Intracavitary Delivery in Mouse and Man......Page 260 13. Clinical and Immunologic Responses to Gene Transfer of an Allogeneic Major Histocompatibility Complex Antigen......Page 278 14. Defective Tumor Suppressor Gene Replacement and Oncogene Inactivation for the Treatment of Cancer......Page 300 15. The Molecular Basis of Bladder Cancer and Prospects for Gene Therapy Using Hammerhead Ribozymes......Page 316 l6. In Situ Gene Insertion for Immunotherapy Using Vaccinia Virus Vectors......Page 332 17. Antisense Oligonucleotide-Based Therapy for HIV-1 Infection from Laboratory to Clinical Trials......Page 352 18. Treatment of Retinitis Induced by Cytomegalovirus Using Intravitreal Fomivirsen (Isis 2922)......Page 374 19· Synthetic DNA-Based Compounds for the Prevention of Coronary Restenosis: Current Status and Future Challenges......Page 384 20. Prevention of Restenosis by Gene Targeting......Page 416 Index......Page 434 An important new collection of clinical and preclinical reports on genetic therapy, this book describes illustrative examples of diseases in which gene-based interventions are presently plausible, and presents case studies of current research using both synthetic oligonucleotides and biological vectors. Combining the insights of over 50 contributors, Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors furnishes a historical overview of genetic therapy highlights official Food and Drug Administration positions on the preparation of oligonucleotides and vectors offers practical models of agent preparation, animal testing, pharmacokinetics, toxicology, and clinical trials discusses both synthetic DNA and biological vector approaches to cancer, viral, and cardiological indications illustrates for new practitioners how each stage of genetic therapy is developed details genetic treatment of leukemia; lymphoma; cancer of the brain, breast, colon, kidney, and lung; melanoma; HIV; and coronary restenosis includes examples of antisense, ribozyme, tumor suppressor, immunostimulation, and gene replacement therapy and addresses questions of preparation, delivery, toxicity, mechanism, and specificity.