Gene Therapy Technologies, Applications and Regulations

Anthony Meager9780471967095, 0-471-96709-2

Gene Therapy Technologies, Applications and Regulations From Laboratory to Clinic Edited by Anthony Meager Division of Immunobiology, The National Institute for Biological Standards and Control, South Mimms, UK The development of gene-based technologies has been rapid over the past decade and has consequently resulted in a surge of interest in human gene therapy, the deliberate transfer of genes to somatic cells to cure or alleviate disease symptoms. Hundreds of clinical protocols involving variously designed vectors for efficient gene transfer have been developed. However, the use of such complex ‘gene medicines’ containing potentially heritable genes has raised numerous concerns regarding quality, efficacy and safety. Encompassing recent developments in the field and addressing current concerns this book: * surveys many of the current technologies for preparing vectors for use in gene therapy protocols * reviews the application of gene-mediated therapies to a range of medical conditions * considers the regulatory aspects of gene therapy including product quality and safety requirements * appraises the transfer of technologies from laboratory to clinic with regard to the attendant requirements and facilities for: * good laboratory practice (GLP) conditions in the R&D laboratory * large-scale production methods and good manufacturing practice (GMP) * current in-process and final product testing Written by international experts knowledgeable about many aspects of human somatic gene therapy, this book will be an essential guide for those embarking on gene therapy technologies relevant to specifications of production and testing of products (and procedures) required to meet existing regulations, including quality, efficacy and safety considerations.

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Table of contents :
Contents……Page 5
Preface……Page 20
1 Cationic Liposomes for Gene Therapy Applications……Page 26
2 DNA Condensation and Receptor-mediated Gene Transfer……Page 38
3 Retroviral Vectors……Page 60
4 Lentiviral Vectors……Page 86
5 Adenoviral Vectors……Page 112
6 Adeno-Associated Viral Vectors……Page 133
7 Advances in Engineering HSV Vectors for Gene Transfer to the Nervous System……Page 150
8 Mammalian Artificial Chromosomes: Prospects for Gene Therapy……Page 187
9 Gene Therapy for Severe Combined Immunodeficiency……Page 201
10 Gene Therapy for Haemophilia……Page 216
11 Cystic Fibrosis: Gene Therapy Approaches……Page 228
12 Therapeutic Approaches to Haemoglobinopathies……Page 248
13 Gene Therapy Approaches to Duchenne Muscular Dystrophy……Page 263
14 Lysosomal Storage Disorders……Page 287
15 Prospects for Gene Therapy of HIV Infections and AIDS……Page 311
16 The Development of the Regulatory Process in Europe for Biological Medicines: How it Affects Gene Therapy Products……Page 339
17 Development and Regulation of Gene Therapy Drugs in Germany……Page 367
18 The Transfer of Technology from the Laboratory to the Clinic: In Process Controls and Final Product Testing……Page 379
19 Good Laboratory Practice in the Research and Development Laboratory……Page 394
20 Facilities for Large-Scale Production of Vectors under GMP Conditions……Page 401
Addendum……Page 419
Index……Page 420