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Gene Therapy Technologies, Applications and Regulations
From Laboratory to Clinic

Edited by ANTHONY MEAGER, The National Institute for Biological Standards and Control, UK

An authoritative and comprehensive read, this book brings together, for the first time, current data previously separated in health authority and regulatory guidelines, government legislative documents, and primary literature. It combines information on gene therapy technologies and the attendant regulatory requirements for gene therapy products and procedures. With ever-increasing interest in this subject and a lack of authoritative texts to date, this is an important contribution to the field.

Contents:

  • Current Technologies for Vector Production and Transgene Expression: Lipsomes
  • Complexed DNA
  • Retroviral Vectors
  • Adenoviral Vectors
  • Adenovirus Associated Viral Vectors
  • Herpes Viral Vectors
  • Other Viral Vectors
  • Artificial Chromosomes
  • Neo-organs
  • The Potential of Gene Therapy in Clinical Conditions: Severe Combined Immunodeficiency (SCID), Adenosine Deaminase Deficiency
  • Cystic Fibrosis
  • Haemophilias
  • Thalassaemias
  • Duchenne Muscular Dystrophy
  • Lysosomal Storage Disorders
  • Hypercolesterolaemia
  • Cancer
  • AIDS
  • Age-related Diseases: Arthritis, Alzheimer’s, Parkinson’s Disease
  • Autoimmune Diseases: Diabetes, Multiple Sclerosis
  • The Regulatory Aspects of Gene Therapy: Quality Considerations in the Production of Vectors and Genetically Altered Cells
  • Preclinical Safety Testing
  • Environmental Safety Concerns
  • Medical Safety and Ethical Issues
  • The Transfer of Technology from Laboratory to Clinic: Setting up GLP Conditions in the R&D Laboratory
  • Facilities for Large-Scale Production of Vectors Under GMP Conditions
  • In Process Controls, Final Product Testing, QA
  • Clinical Facilities for Product Administration and Monitoring of Patients
  • Future Concerns and Directions: Long-Term Health Considerations for Patients receiving Gene Therapy
  • Germline Gene Therapy

0471 96709 2September 1999approx 438ppHardback

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