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

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Meager, Anthony

Jossey-Bass, Incorporated Publishers

Wiley & Sons, Incorporated, John

John Wiley & Sons, Incorporated

WILEY COMPUTING Publisher

Spectrum Publications

Halsted Press

Chichester, England, New York, England, 1999

John Wiley & Sons, Inc., Chichester, 1999

United States, United States of America

Molecular biology, Chichester, ©1999

October 29, 1999

2001

mexmat -- 22

lg27137

{"edition":"1","isbns":["0470842385","0471967092","9780470842386","9780471967095"],"last_page":438,"publisher":"Wiley"}

Includes bibliographical references and index

Cationic Liposomes For Gene Therapy Applications / J.p. Clancy And Eric S. Sorscher -- Dna Condensation And Receptor-mediated Gene Transfer / Assem Ziady And Thomas Ferkol -- Retroviral Vectors / Walter H. Günzburg And Brian Salmons -- Lentiviral Vectors / Andrew M.l. Lever -- Adenoviral Vectors / Sheila Connelly -- Adeno-associated Viral Vectors / Terence R. Flotte And Barrie J. Carter -- Advances In Engineering Hsv Vectors For Gene Transfer To The Nervous System / M. Karina Soares [and Others] -- Mammalian Artificial Chromosomes : Prospects For Gene Therapy / Tom A. Ebersole And Christine J. Farr -- Gene Therapy For Severe Combined Immunodeficiency / Adrian J. Thrasher, Hubert B. Gaspar And Christine Kinnon -- Gene Therapy For Haemophilia / Rob C. Hoeben [and Others] -- Cystic Fibrosis : Gene Therapy Approaches / Natasha J. Caplen -- Therapeutic Approaches To Haemoglobinopathies / Yves Beuzard -- Gene Therapy Approaches To Duchenne Muscular Dystrophy / Stephen Murphy And George Dickson -- Lysosomal Storage Diseases / Linda S. Lashford, Leslie J. Fairbairn And J.e. Wraith -- Prospects For Gene Therapy Of Hiv Infections And Aids / Caroline G.l. Lee, Michael M. Gottesman And Kuan-teh Jeang -- The Development Of The Regulatory Process In Europe For Biological Medicines : How It Affects Gene Therapy Products / Anthony Meager, Tobias Vocke And Gerd Zimmermann -- Development And Regulation Of Gene Therapy Drugs In Germany / Klaus Cichutek -- The Transfer Of Technology From The Laboratory To The Clinic: In Process Controls And Final Product Testing / Flavia Borellini And Jeffrey M. Ostrove -- Good Laboratory Practice In The Research And Development Laboratory / Alasdair J. Shepherd -- Facilities For Large-scale Production Of Vectors Under Gmp Conditions / Janice E. Boyd. Edited By Anthony Meager. Includes Bibliographical References And Index. Mode Of Access: World Wide Web. System Requirements: Internet Connectivity, World Wide Web Browser, Adobe Acrobat Reader. Html And Pdf Text.

<p><p>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&#58;<br> * Surveys Many Of The Current Technologies For Preparing Vectors For Use In Gene Therapy Protocols<br> * Reviews The Application Of Gene-mediated Therapies To A Range Of Medical Conditions<br> * Considers The Regulatory Aspects Of Gene Therapy Including Product Quality And Safety Requirements<br> * Appraises The Transfer Of Technologies From Laboratory To Clinic With Regard To The Attendant Requirements And Facilities For&#58;<br> * Good Laboratory Practice (glp) Conditions In The R&d Laboratory<br> * Large-scale Production Methods And Good Manufacturing Practice (gmp)<br> * Current In-process And Final Product Testing<br> <br> 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.</p>

Considered an extremely promising area of biomedical research, gene therapy has the potential to treat diseases both inherited and acquired (e.g., cystic fibrosis and cancer), and may eventually complement or replace conventional drug therapy. This reference presents up-to-date information on gene therapy technologies as well as the regulatory requirements for gene therapy products and procedures. It brings together in one volume data previously separated in health authority and regulatory guidelines, government legislative documents, and the primary literature.

This text brings together the current data previously separated. It combines information on gene therapy technologies and the attendant regulatory requirements for gene therapy, products and procedures

2009-07-20