Gene Therapy Technologies, Applications and Regulations: From Laboratory to Clinic

Description

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.Content: Chapter 1 Cationic Liposomes for Gene Therapy Applications (pages 1–12): J.P. Clancy and Eric S. SorscherChapter 2 DNA Condensation and Receptor?mediated Gene Transfer (pages 13–34): Assem Ziady and Thomas FerkolChapter 3 Retroviral Vectors (pages 35–60): Walter H. Gunzburg and Brian SalmonsChapter 4 Lentiviral Vectors (pages 61–86): Andrew M.L. LeverChapter 5 Adenoviral Vectors (pages 87–107): Sheila ConnellyChapter 6 Adeno?Associated Viral Vectors (pages 109–125): Terence R. Flotte and Barrie J. CarterChapter 7 Advances in Engineering HSV Vectors for Gene Transfer to the Nervous System (pages 127–163): M. Karina Soares, William F. Goins, Joseph C. Glorioso and David J. FinkChapter 8 Mammalian Artificial Chromosomes: Prospects for Gene Therapy (pages 165–178): Tom A. Ebersole and Christine J. FarrChapter 9 Gene Therapy for Severe Combined Immunodeficiency (pages 179–193): Adrian J. Thrasher, Hubert B. Gaspar and Christine KinnonChapter 10 Gene Therapy for Haemophilia (pages 195–206): Rob C. Hoeben, Frederik H.E. Schagen, Marjolijn M. van der Eb, Frits J. Fallaux, Alex J. van der Eb and Hans van OrmondtChapter 11 Cystic Fibrosis: Gene Therapy Approaches (pages 207–226): Natasha J. CaplenChapter 12 Therapeutic Approaches to Haemoglobinopathies (pages 227–241): Yves BeuzardChapter 13 Gene Therapy Approaches to Duchenne Muscular Dystrophy (pages 243–266): Stephen Murphy and George DicksonChapter 14 Lysosomal Storage Disorders (pages 267–290): Linda S. Lashford, Leslie J. Fairbairn and J.E. WraithChapter 15 Prospects for Gene Therapy of HIV Infections and AIDS (pages 291–318): Caroline G.L. Lee, Michael M. Gottesman and Kuan?Teh JeangChapter 16 The Development of the Regulatory Process in Europe for Biological Medicines: How it Affects Gene Therapy Products (pages 319–346): Anthony Meager, Tobias Vocke and Gerd ZimmermannChapter 17 Development and Regulation of Gene Therapy Drugs in Germany (pages 347–358): Klaus CichutekChapter 18 The Transfer of Technology from the Laboratory to the Clinic: In Process Controls and Final Product Testing (pages 359–373): Flavia Borellini and Jeffrey M. OstroveChapter 19 Good Laboratory Practice in the Research and Development Laboratory (pages 375–381): Alasdair J. ShepherdChapter 20 Facilities for Large?Scale Production of Vectors under GMP Conditions (pages 383–400): Janice E. Boyd