Nonviral Vectors for Gene Therapy

1. Front Matter

   Pages i-xi

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2. Synthesis of Polyampholyte Comb-Type Copolymers Consisting of a Poly(L-lysine) Backbone and Hyaluronic Acid Side Chains for DNA Carrier

   • Atsushi Maruyama, Yoshiyuki Takei

   Pages 1-9

3. Cationic α-Helical Peptides for Gene Delivery into Cells

   • Takuro Niidome, Haruhiko Aoyagi

   Pages 11-21

4. Supramolecular Self-Assembly of Poly(ethylene glycol)-Block-Poly (L-lysine) Dendrimer with Plasmid DNA

   • Joon Sig Choi, Jong Sang Park

   Pages 23-33

5. Water-Soluble Cationic Methacrylate Polymers for Nonviral Gene Delivery

   • Gert W. Bos, Daan J. A. Crommelin, Wim E. Hennink

   Pages 35-60

6. Stabilization of Polycation-DNA Complexes by Surface Modification with Hydrophilic Polymers

   • David Oupicky, Martin L. Read, Thierry Bettinger

   Pages 61-78

7. Use of Disulfide Cationic Lipids in Plasmid DNA Delivery

   • Fuxing Tang, Jeffrey A. Hughes

   Pages 79-88

8. Interactions of Lipid-Oligonucleotide Conjugates with Low-Density Lipoprotein

   • Erik T. Rump, Erik A. L. Biessen, Theo J. C. van Berkel, Martin K. Bijsterbosch

   Pages 89-104

9. Coupling of Nuclear Localization Signals to Plasmid DNA

   • Carole Neves, Daniel Scherman, Pierre Wils

   Pages 105-109

10. Progress Toward a Synthetic Virus

    • Bo-Hua Zhong, George Y. Wu, Catherine H. Wu

    Pages 111-121

11. Characterization of Polyampholyte Comb-Type Copolymer DNA Carriers

    • Yoshiyuki Takei, Atsushi Maruyama, Toshihiro Akaike, Nobuhiro Sato

    Pages 123-129

12. Methods for Studying the Formation of Polycation-DNA Complexes and Properties Useful for Gene Delivery

    • Martin L. Read, Thierry Bettinger, David Oupicky

    Pages 131-148

13. Characterization of DNA Condensates by Atomic Force Microscopy

    • Ye Fang, Jan H. Hoh

    Pages 149-158

14. Rapid and Systematic Transfer and Recovery of Large BACs/PACs into Mammalian Cells by HAEC Retrofitting

    • Rona S. Scott, Jean-Michel H. Vos

    Pages 159-173

15. Systemic Delivery of Therapeutic Proteins by Intramuscular Injection of Plasmid DNA

    • Holly M. Horton, Suezanne E. Parker

    Pages 175-183

16. Local Delivery of Therapeutic Proteins by Intratumoral Injection of Plasmid DNA-Lipid Complexes

    • Holly M. Horton, Suezanne E. Parker

    Pages 185-193

17. Nonviral DNA Delivery from Polymeric Systems

    • Lonnie D. Shea, David J. Mooney

    Pages 195-207

18. Promotion of Duplex and Triplex DNA Formation by Polycation Comb-Type Copolymers

    • Hidetaka Torigoe, Atsushi Maruyama

    Pages 209-224

19. Lyophilization of Nonviral Gene Delivery Systems

    • S. Dean Allison, Thomas J. Anchordoquy

    Pages 225-252

20. Ultraviolet Absorption and Circular Dichroism Spectroscopy of Nonviral Gene Delivery Complexes

    • Chad S. Braun, Lisa A. Kueltzo, C. Russell Middaugh

    Pages 253-284

The purpose of this volume of Methods in Molecular Medicine is to set forth examples of the great variety of techniques and applications that are now emerging in the field of nonviral gene therapy. The book emphasizes not only specific approaches to gene delivery but, in particular, the best current me- ods to prepare, handle, and characterize gene delivery agents. These topics are of very broad importance since gene therapy evolves from its mostly ac- emy-based experimental and clinical research to the ever increasing number of industry-driven programs directed toward commercial development. S- cessful introduction of nonviral gene therapy agents into the clinic should be expected to require rigorous manufacturing and analytical methods that readily meet the regulatory guidelines under which new drug candidates are reviewed for marketing approval. Exactly what those guidelines will prove to be c- tainly depends on the established guidelines for review of both biological and chemical therapeutics. Additionally, many new techniques are being devised and applied to gene therapy research; these techniques will be instrumental in developing and characterizing successful gene delivery agents. Nonviral Vectors for Gene Therapy: Methods and Protocols has two main sections. To start with, there is a series of chapters on specific protocols for the synthesis, characterization, and application of gene delivery agents. S- eral chapters address the topic of materials to bind with DNA to form the compact condensed phases that facilitate cellular delivery.

"The book begins with a series of chapters covering specific protocols concerning gene delivery agents. These are followed by several review chapters that provide additional information on preparing and characterizing gene transfer agents. Particular focus is given to spectroscopic techniques. The final chapter provides a review of renal gene therapy. Chapters are well organized and often accompanied by helpful figures. Each chapter includes a list of references, an an index is provided. Contributors are from research facilities throughout the world. This book is highly recommended for collections serving researchers in the area of gene therapy." - E-Streams

• Praecis Pharmaceuticals Incorporated, Waltham

  Mark A. Findeis

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