Description
Book SynopsisGene 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 medic
Trade Review"It succeeds in putting together, possibly for the first time, gene therapy vectors, clinical applications, and regulatory production issues." --Human Genetics, April 2000
Table of ContentsCationic Liposomes for Gene Therapy Applications (J. Clancy & E. Sorscher).
DNA Condensation and Receptor-mediated Gene Transfer (A. Ziady & T. Ferkol).
Retroviral Vectors (W. Günzburg & B. Salmons).
Lentiviral Vectors (A. Lever).
Adenoviral Vectors (S. Connelly).
Adeno-associated Viral Vectors (T. Flotte & B. Carter).
Advances in Engineering HSV Vectors for Gene Transfer to the Nervous System (M. Soares, et al.).
Mammalian Artificial Chromosomes: Prospects for Gene Therapy (T. Ebersole & C. Farr).
Gene Therapy for Severe Combined Immunodeficiency (A. Thrasher, et al.).
Gene Therapy for Haemophilia (R. Hoeben, et al.).
Cystic Fibrosis: Gene Therapy Approaches (N. Caplen).
Therapeutic Approaches to Haemoglobinopathies (Y. Beuzard).
Gene Therapy Approaches to Duchenne Muscular Dystrophy (S. Murphy & G. Dickson).
Lysosomal Storage Diseases (L. Lashford, et al.).
Prospects for Gene Therapy of HIV Infections and AIDS (C. Lee, et al.).
The Developemnt of the Regulatory Process in Europe for Biological Medicines: How it Affects Gene Therapy Products (A. Meager, et al.).
Developement and Regulation of Gene Therapy Drugs in Germany (K. Cichutek).
The Transfer of Technology from the Laboratory to the Clinic: In Process Controls and Final Product Testing (F. Borellini & J. Ostrove).
Good Laboratory Practice in the Research and Development Laboratory (A. Shepherd).
Facilities for Large-scale Production of Vectors Under GMP Conditions (J. Boyd).
Addendum.
Index.
