Non Viral Gene Therapy

Presents information on non-viral gene-delivery techniques, covering a spectrum of disciplines that include chemistry, molecular biology, cell biology, and pharmacokinetics. This work is useful to researchers and engineers in genetic engineering, molecular medicine, biochemical engineering, and biotechnology.

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.

Gene transfer within humans has been an obstacle until about 10 years ago. At that time, it was found that viral vectors were effective carriers of "healthy genes" into patients' cells. The problem, however, was that viral vectors proved unnecessarily harmful to humans: subjects experienced inflamatory activity and negative immunological responses to the genes. Viral vectors were also unable to meet the needs of the pharmaceutical community: they were not reproducible in large-scale proportions in cost-effective ways. Thus, research was undertaken to find a safer way to transfer genes to patients without jeopardizing the safety of the patient. And so non-viral vectors were discovered. This volume presents the various non-viral vectors currently under development. Although not methodologically oriented, it will provide the necessary details behind the development of the vectors. This information will prove useful to both researchers and clinicians. Key Features * Presents state-of-the art developments of nonviral vectors as tools for modern molecular medicine * Covers all types of nonviral vectors, from molecular structure to therapeutic application Provides a comprehensive review of synthetic vectors * Includes contributions from major investigators and leading experts in the field

The field of non-viral vector research has rapidly progressed since the publication of the first edition. This new edition is expanded to two separate volumes that contain in-depth discussions of different non-viral approaches, including cationic liposomes and polymers, naked DNA and various physical methods of delivery, as well as a comprehensive coverage of the molecular biological designs of the plasmid DNA for reduced toxicity, prolonged expression and tissue or disease specific genes. New developments such as the toxicity of the non-viral vectors and recent advances in nucleic acid therapeutics are fully covered in these volumes.

Polymers and Nanomaterials for Gene Therapy provides the latest information on gene therapy, a topic that has attracted significant attention over the past two decades for the treatment of inherited and acquired genetic diseases. Major research efforts are currently focused on designing suitable carrier vectors that compact and protect oligonucleotides for gene therapy. The book explores the most recent developments in the field of polymer science and nanotechnology, and how these advancements have helped in the design of advanced materials. Non-viral vector systems, including cationic lipids, polymers, dendrimers, peptides and nanoparticles, are potential routes for compacting DNA for systemic delivery. However, unlike viral analogues that have no difficulty in overcoming cellular barriers and immune defense mechanisms, non-viral gene carriers consistently exhibit significant reduced transfection efficiency due to numerous extra- and intracellular obstacles. Therefore, biocompatibility and potential for large-scale production make these compounds increasingly attractive for gene therapy. This book contains chapters on the engineering of polymers and nanomaterials for gene therapy, and how they can form complexes with DNA and avoid both in vitro and in vivo barriers. Other chapters describe in vitro, ex vivo, in vivo gene therapy studies, and the current issues affecting non-viral gene therapy. Explores current challenges in the research of genetic diseases Discusses polymers for gene therapy and their function in designing advanced materials Provides examples of organic and inorganic nanomaterials for gene therapy Includes labeling, targeting, and assays Looks at characterization, physico-(bio)chemical properties, and applications

Annotation The field of non-viral vector research has rapidly progressed since the publication of the first edition. This new edition is expanded to two separate volumes that contain in-depth discussions of different non-viral approaches, including cationic liposomes and polymers, naked DNA and various physical methods of delivery, as well as a comprehensive coverage of the molecular biological designs of the plasmid DNA for reduced toxicity, prolonged expression and tissue or disease specific genes. New developments such as the toxicity of the non-viral vectors and recent advances in nucleic acid therapeutics are fully covered in these volumes.

The field of genetics is rapidly evolving, and new medical breakthroughs are occurring as a result of advances in our knowledge of genetics. Advances in Genetics continually publishes important reviews of the broadest interest to geneticists and their colleagues in affiliated disciplines. Includes methods for testing with ethical, legal, and social implications Critically analyzes future directions Written and edited by recognized leaders in the field

Provides expert, state-of-the-art insight into the currentprogress of viral and non-viral gene therapy Translational medicine has opened the gateway to the era ofpersonalized or precision medicine. No longer a one-size-fits-allapproach, the treatment of cancer is now based on an understandingof underlying biologic mechanisms and is increasingly beingtailored to the molecular specificity of a tumor. This book provides a comprehensive overview of the pertinentmolecular discoveries in the cancer field and explains how theseare being used for gene-based cancer therapies. Designed as avolume in the Translational Oncology book series, Cancer GeneTherapy by Viral and Non-viral Vectors deals with the practiceof gene-therapy, with reference to vectors for gene expression andgene transfer, as well as viral therapy. It covers the history andcurrent and future applications of gene transfer in cancer, andprovides expert insight on the progress of viral and non-viral genetherapy with regard to delivery system, vector design, potentialtherapeutic genes, and principles and regulations for cancer genetherapy. Presented in three parts, Cancer Gene Therapy by Viral andNon-viral Vectors covers: Delivery Systems • Translational Cancer Research: Gene Therapy by Viraland Non-viral Vectors • Retroviruses for Cancer Therapy • DNA Plasmids for Non-viral Gene Therapy ofCancer • Cancer Therapy with RNAi delivered by Non-viralMembrane/Core Nanoparticles Targeted Expression • Cancer Gene Therapy by Tissue-specific andCancer-targeting Promptors • MicroRNAs as Drugs and Drug Targets in Cancer Principles of Clinical Trials in Gene Therapy • Regulatory issues for Manufacturers of Viral Vectorsand Vector-transduced Cells for Phase I/II Trials • US Regulations Governing Clinical Trials in GeneTherapy • Remaining Obstacles to the Success of Cancer GeneTherapy Focusing on speeding the process in clinical cancer care bybringing therapies as quickly as possible from bench to bedside,Cancer Gene Therapy by Viral and Non-viral Vectors is anabsolutely vital book for physicians, clinicians, researchers, andstudents involved in this area of medicine.