Nonviral Vectors for Gene Therapy: Methods and Protocols (Methods in Molecular Medicine, 65)

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.

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.