Nonviral Vectors for Gene Therapy: Methods and Protocols

ISBN-13: 9781617371455 / Angielski / Miękka / 2010 / 399 str.

Mark A. Findeis

Opis

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.

Recenzje

"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

Spis treści

Synthesis of Polyampholyte Comb-Type Copolymers Consisting of a Poly(l-lysine) Backbone and Hyaluronic Acid Side Chains for DNA Carrier Atsushi Maruyama and Yoshiyuki Takei Cationic _-Helical Peptides for Gene Delivery into Cells Takuro Niidome and Haruhiko Aoyagi Supramolecular Self-Assembly of Poly(ethylene glycol)-Block-Poly (l-lysine) Dendrimer with Plasmid DNA Joon Sig Choi and Jong Sang Park Water-Soluble Cationic Methacrylate Polymers for Nonviral Gene Delivery Gert W. Bos, Daan J. A. Crommelin, and Wim E. Hennink Stabilization of Polycation-DNA Complexes by Surface Modification with Hydrophilic Polymers David Oupicky, Martin L. Read, and Thierry Bettinger Use of Disulfide Cationic Lipids in Plasmid DNA Delivery Fuxing Tang and Jeffrey A. Hughes Interactions of Lipid-Oligonucleotide Conjugates with Low-Density Lipoprotein Erik T. Rump, Erik A. L. Biessen, Theo J. C. van Berkel, and Martin K. Bijsterbosch Coupling of Nuclear Localization Signals to Plasmid DNA Carole Neves, Daniel Scherman, and Pierre Wils Progress Toward a Synthetic Virus: A Multicomponent System for Liver-Directed DNA Delivery Bo-Hua Zhong, George Y. Wu, and Catherine H. Wu Characterization of Polyampholyte Comb-Type Copolymer DNA Carriers Yoshiyuki Takei, Atsushi Maruyama, Toshihiro Akaike, and Nobuhiro Sato Methods for Studying the Formation of Polycation-DNA Complexes and Properties Useful for Gene Delivery Martin L. Read, Thierry Bettinger, and David Oupicky Characterization of DNA Condensates by Atomic Force Microscopy Ye Fang and Jan H. Hoh Rapid and Systematic Transfer and Recovery of Large BACs/PACs into Mammalian Cells by HAEC Retrofitting Rona S. Scott and Jean-Michel H. Vos Systemic Delivery of Therapeutic Proteins by Intramuscular Injection of Plasmid DNA Holly M. Hortonand Suezanne E. Parker Local Delivery of Therapeutic Proteins by Intratumoral Injection of Plasmid DNA-Lipid Complexes Holly M. Horton and Suezanne E. Parker Nonviral DNA Delivery from Polymeric Systems Lonnie D. Shea and David J. Mooney Promotion of Duplex and Triplex DNA Formation by Polycation Comb-Type Copolymers Hidetaka Torigoe and Atsushi Maruyama Lyophilization of Nonviral Gene Delivery Systems S. Dean Allison and Thomas J. Anchordoquy Ultraviolet Absorption and Circular Dichroism Spectroscopy of Nonviral Gene Delivery Complexes Chad S. Braun, Lisa A. Kueltzo, and C. Russell Middaugh Characterization of Synthetic Gene Delivery Vectors by Infrared Spectroscopy Sirirat Choosakoonkriang, Christopher M. Wiethoff, Lisa A. Kueltzo, and C. Russell Middaugh Characterization of Cationic Vector-Based Gene Delivery Vehicles Using Isothermal Titration and Differential Scanning Calorimetry Brian A. Lobo, Sheila A. Rogers, Christopher M. Wiethoff, Sirirat Choosakoonkriang, Susan Bogdanowich-Knipp, and C. Russell Middaugh Light-Scattering Techniques for Characterization of Synthetic Gene Therapy Vectors Christopher M. Wiethoff and C. Russell Middaugh Renal Gene Therapy Yeong-Hau H. Lien and Li-Wen Lai

Blurb

Nonviral gene therapy-an emerging field with great clinical promise-avoids many problems associated with viral gene therapy and offers the possibility of superior clinical effectiveness. In Nonviral Vectors for Gene Therapy: Methods and Protocols, Mark A. Findeis has assembled a panel of active researchers to present their best methods not only for preparing, handling, and characterizing gene delivery agents, but also for gene delivery. To help those preparing and characterizing gene transfer agents, the contributors examine a broad range of compounds that bind with DNA to form the compact complexes that facilitate cellular delivery-among them peptide conjugates, synthetic polymers, and lipids. They also outline specific approaches to gene transfer in vivo, including direct delivery by intratumoral injection and indirect delivery by cell-specific targeting of DNA complexes, and discuss in detail many spectroscopic techniques for characterizing nonviral gene delivery agents. Opportunities for the development and application of these novel vectors in the research lab and eventually, in the clinic, are highlighted.
Comprehensive and state-of-the-art, Nonviral Vectors for Gene Therapy: Methods and Protocols illuminates for today's investigators the powerful new approaches to the creation of nonviral materials, as well as their efficacious use in delivering therapeutic genes to patients across the spectrum of human disease.