DNA-Pharmaceuticals —— Formulation and Delivery in Gene Therapy, DNA Vaccination and Immunotherapy

----- DNA-药物：基因治疗与DNA疫苗的配方与送药

ISBN: 9783527311873 出版年：2006 页码：277 Martin Schleef Wiley

Preface. List of Contributors. Abbreviations. 1. DNA Vaccines - An Overview (Britta Wahren and Margaret Liu). 1.1 Rational for DNA Vaccines. 1.2 Preclinical Proof of Concept. 1.3 Clinical trials. 1.4 Second-Generation Vaccines. 1.5 Conclusions. 2. DNA as a Pharmaceutical - Regulatory Aspects (Carsten Kneuer). 2.1 Introduction. 2.2 Quality Requirements for DNA used as a Gene Therapy Product. 2.3 Safety Studies for Clinical Trials. 2.4 Special Issues. 2.5 Biosafety Issues and Environmental Risk Assessment. 3. From Bulk to Delivery: Plasmid Manufacturing and Storage (Carsten Vos, Torsen Schmidt and Martin Schleef). 3.1 Introduction. 3.2 Manufacturing of Plasmid DNA. 3.3 Quality Control of Plasmid DNA Vectors. 3.4 Plasmid Topology (Structural Homogeneity). 3.5 Future Developments. 4. Minimized, CpG-Depleted, and Methylated DNA Vectors: Toward Perfection in Nonviral Gene Therapy (Oleg Tolmachov, Richard Harbottle, Brian Bigger and Charles Coutelle). 4.1 Introduction. 4.2 The Mammalian Immune System as a Barrier to Nonviral Gene Delivery. 4.3 Strategies of Minimize DNA Vectors. 4.4 Depletion of CpG Dinucleotides on the Bacterial Vector Backbone. 4.5 Methylation of CpG Dinucleotides in Plasmid DNA. 4.6 Towards an Ideal Nonviral Vector. 4.7 Conclusion. 5. Localized Nucleic Acid Delivery: A Discussion of Selected Methods (Christian Plank, Franz Scherer and Carsten Rudolph). 5.1 Foreword. 5.2 Nucleic Acid Delivery - What For? 5.3 Nucleic Acid Delivery - How? 5.4 Why Is Localization of Drug and Nucleic Acid Delivery Important? 5.5 Hierarchies of Localization (Targeting). 5.6 Concluding Remarks. 6. DNA Needle Injection (Matthias Giese). 6.1 From Mouse to Human. 6.2 Intramuscular Injection. 6.3 Intradermal Injection. 6.4 Concluding Remarks. 7. Needleless Jet Injection of Naked DNA for Nonviral in vivo Gene transfer (Wolfgang Walther and Ulrike Stein). 7.1 Introduction. 7.2 In vivo Application of Jet Injection. 7.3 Conclusions. 8. Plasmid Inhalation: Delivery to the Airways (Lee A. Davies, Stephen C. Hyde and Deborah R. Gill). 8.1 Introduction. 8.2 Delivery Methods. 8.3 Future Directions. 8.4 Conclusions. 9. Hydrodynamic Gene Delivery (John W. Fabre). 9.1 Definition. 9.2 Initial Discovery of the Technique. 9.3 The Systemic Hydrodynamic Approach. 9.4 The Regional Hydrodynamic Approach to the Liver. 9.5 Gene Delivery to the Liver in Large Animals. 9.6 Hydrodynamic Gene Delivery to Tissues Other Than Liver. 9.7 Mechanisms of Gene Delivery. 9.8 Safety and Clinical Applicability. 10. DNA Pharmaceuticals for Skin Diseases (Vitali Alexeev and Jouni Utto). 10.1 Introduction. 10.2 Recombinant DNA-Based Skin Gene Therapy. 10.3 DNA Vaccines. 10.4 Physical Methods of DNA Delivery. 11. Electrotransfection - An Overview (Capucine Trollet, Pascal Bigey and Daniel Scherman). 11.1 Theory and Mechanisms. 11.2 In vivo DNA Elctrotransfer in Practice. 11.3 Targeted Tissues. 11.4 Therapeutic Applications. 11.5 Conclusion. 12. Electrogenetransfer in Chemical Applications (Lluis M. Mir). 12.1 Summary of the Basis of Electrogenetherapy. 12.2 The Road to Clinical Electrogenetherapy. 13. Cancer Inhibition in Mice After Systemic Application of Plasmid-Driven Expression of Small Interfering RNAs (Birgit Spankuch and Klaus Strebhardt). 13.1 Introduction. 13.2 Plasmid-Expressed siRNA. 13.3 Conclusion and Future Directions. Subject Index.