Concepts in Genetic Medicine

by Editor: Boro Dropulic (VIRxSYS, USA ); Editor: Barrie Carter (Targeted Genetics, USA )

• ISBN13: 9780470184578
• ISBN10: 0470184574
• Format: eBook
• Copyright: 2008-01-01
• Publisher: Wiley

Summary

This book delivers a collection of organized and succinct reviews in the field of therapeutic genetic medicine presented by a carefully selected group of top experts. Each chapter focuses on a single, current topic and explains issues in the development of genetic treatments, critical challenges and strategies for implementing them, and future directions in research and translational applications. The book is complemented with uniformly designed illustrations and a supplementary Web site with a PowerPoint presentation for educators.

Table of Contents

Introduction

The Evolution of Human Gene Therapy. A Journey from Excessive Hype to Excessive Diffidence to Realty

Delivery Systems

Murine Leukemia Virus Based Vectors

Lentivirus Vectors

Adenoviral Vectors: History and Perspective

Adeno-associated Virus Vectors

The Glass is Half Full: the Goal of Long-term delivery may be Approachable Using SV40-derived Vectors

Herpes Simplex Virus Vectors

Nonviral Gene Delivery Systems

Applications

Therapeutic Gene Transfer to Skeletal Muscle

Gene Therapy for Cardiovascular Disease

Intra-articular Vector Delivery for Inflammatory Disease

The Respiratory System as a Platform for Gene Delivery

The Brain as a Target for Gene Therapy

Immune Responses to Viral vectors Injected Systemically or into the CNS

Cancer vaccines

Genetically Modified T cells for Human Gene Therapy

Lentiviral Vector Delivery of RNAi Triggers for the Treatment of HIV-1 Infection

Manufacture

The Manufacture of Genetic Viral Vector Products

The Manufacture of Adeno-associated Viral Vectors

Lentivirus Vector Manufacturing

Safety and Regulatory

Assays for the Release of Viral Vector Gene Therapy Products for Clinical Trials

Safety of Retroviral Vectors: Regulatory and Technical Considerations

Assays for the Quality Control of Lentiviral Vectors

Assays for Non-viral vectors

Assays for the Release of Cellular Gene Therapy Products

Toxicology for Gene Therapy Products: Concepts and Challenges

Regulatory Aspects of Gene Therapy Medicinal Products in the European Union

Finance and Reimbursment

Venture Capital and Biotechnology Startups

The role of Investment Banking in the Growth of Biotechnology Companies

Managing Reimbursment for Gene Therapy Products

Table of Contents provided by Publisher. All Rights Reserved.