TY  - BOOK
AU  - Dropulic,Boro
AU  - Carter,Barrie
TI  - Concepts in genetic medicine 
SN  - 9780470184585
PY  - 2008///
CY  - Hoboken, N.J.
PB  - Wiley-Liss
KW  - Medical genetics
KW  - Gene therapy
KW  - Genetic vectors
KW  - Genetic Therapy
KW  - methods
KW  - Gene Transfer Techniques
KW  - Genetic Vectors
KW  - Viruses
KW  - genetics
KW  - Genetics, Medical
N1  - Includes bibliographical references and index; Cover -- TOC36;CONTENTS -- CONTRIBUTORS -- PREFACE -- INTRODUCTION -- CH36;1 The Evolution of Human Gene Therapy58; A Journey from Excessive Hype to Excessive Diffidence to Reality -- VECTOR SYSTEMS -- CH36;2 Murine Leukemia Virus8211;Based Retroviral Vectors -- CH36;3 Lentivirus Vectors -- CH36;4 Adenoviral Vectors58; History and Perspective -- CH36;5 Adeno45;Associated Virus Vectors -- CH36;6 SV40 Virus8211;Derived Vectors -- CH36;7 Herpes Simplex Virus Vectors -- CH36;8 Nonviral Gene Delivery Systems -- APPLICATIONS -- CH36;9 Therapeutic Gene Transfer to Skeletal Muscle -- CH36;10 Gene Therapy for Cardiovascular Disease -- CH36;11 Intraarticular Vector Delivery for Inflammatory Joint Disease -- CH36;12 The Respiratory System as a Platform for Gene Delivery -- CH36;13 The Brain as a Target for Gene Therapy -- CH36;14 Immune Responses to Viral Vectors Injected Systemically or into the CNS -- CH36;15 Cancer Vaccines -- CH36;16 Genetically Modified T Cells for Human Gene Therapy -- CH36;17 Lentiviral Vector Delivery of RNAi for the Treatment of HIV45;1 Infection -- CH36;18 Vector Targeting -- THE MANUFACTURE OF GENE THERAPY PRODUCTS -- CH36;19 The Manufacture of Genetic Viral Vector Products -- CH36;20 The Manufacture of Adeno45;Associated Viral Vectors -- CH36;21 Lentivirus Vector Manufacturing -- SAFETY AND REGULATORY CONSIDERATIONS -- CH36;22 Assays for the Release of Viral Vector Gene Therapy Products for Human Clinical Trials -- CH36;23 Safety of Retroviral Vectors58; Regulatory and Technical Considerations -- CH36;24 Assays for the Quality Control of Lentiviral Vectors -- CH36;25 Assays for Nonviral Vectors -- CH36;26 Assays for the Release of Cellular Gene Therapy Products -- CH36;27 Toxicology for Gene Therapy Products58; Concepts and Challenges -- CH36;28 Regulatory Aspects of Gene Therapy Medicinal Products in the European Union -- FINANCE AND REIMBURSMENT -- CH36;29 Venture Capital and Biotechnology Startups -- CH36;30 The Role of Investment Banking in the Growth of Biotechnology Companies -- CH36;31 Managing Reimbursement for Gene Therapy Products -- IDX36;INDEX -- Last Page
N2  - A collection of reviews that highlight the most important issues in the development, manufacture, and commercialization of novel genetic medicines While the field of gene therapy is still a relatively young discipline, it is rapidly evolving and is characterized by impressive scientific and medical innovation. In Concepts in Genetic Medicine, readers are offered a collection of uniformly 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. Beginning with an introduction to the genetic basis of disease and a discussion of specific diseases, their etiology, and promising therapeutic treatments, the book goes on to cover vector design, genetic payloads and disease targets, vaccine vector systems, manufacture, assays, cell systems and target issues, preclinical testing, regulatory considerations, clinical evaluation, safety concerns, and commercialization. Complete with uniformly designed illustrations and a supplementary Web site with a PowerPoint presentation for educators, this is a practical guide for physicians, scientists, allied health workers, and patient advocacy professionals attempting to develop genetic medicines for translation into the clinic
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