Gene Therapy

  • Mauro¬†Giacca

Table of contents

  1. Front Matter
    Pages i-xxi
  2. Mauro Giacca
    Pages 1-7
  3. Mauro Giacca
    Pages 9-45
  4. Mauro Giacca
    Pages 47-137
  5. Mauro Giacca
    Pages 139-281
  6. Mauro Giacca
    Pages 283-292
  7. Back Matter
    Pages 293-303

About this book


This book presents to the reader a comprehensive and detailed, yet easily accessible, description of the whole field of gene therapy. It covers topics ranging from vector development to the results of the most recent clinical trials. It includes descriptions of lentiviral and AAV vector development, of therapeutic gene selection (including siRNAs, shRNAs, and microRNAs), and of the most recent clinical applications of gene therapy for diseases of the eye, the cardiovascular system, and the central nervous system. Of note, the book only considers conditions for which transfer of genetic material has already progressed to clinical applications or is close to doing so. The language is plain and, whenever possible, non-technical. Since the book is intended to be a textbook in the field of gene therapy in both the basic science and clinical fields, whenever technical descriptions are required these are provided. Readers will include graduate students in the life sciences area in their final years of study, PhD students, and medical doctors specializing in different fields of internal and specialist medicine, in addition to research scientists working in various areas of current biomedical research.


Clinical trials Gene therapy Inherited disorders Molecular genetics Viral vectors genes genome

Authors and affiliations

  • Mauro¬†Giacca
    • 1
  1. 1.International Centre for Genetic Engineering and Biotechnology (ICGEB)TriesteItaly

Bibliographic information