Clinical trials of genetic therapy with antisense DNA and DNA vectors

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