Lentiviral Vectors and Gene Therapy

  • David Escors
  • Karine Breckpot
  • Frederick Arce
  • Grazyna Kochan
  • Holly Stephenson

Part of the SpringerBriefs in Biochemistry and Molecular Biology book series (BRIEFSBIOCHEM)

Table of contents

  1. Front Matter
    Pages i-ix
  2. David Escors, Karine Breckpot
    Pages 1-10
  3. David Escors, Grazyna Kochan, Holly Stephenson, Karine Breckpot
    Pages 11-28
  4. David Escors, Grazyna Kochan, Holly Stephenson, Karine Breckpot
    Pages 29-50
  5. Frederick Arce, Karine Breckpot, Grazyna Kochan, David Escors
    Pages 51-67
  6. Grazyna Kochan, David Escors, Holly Stephenson, Karine Breckpot
    Pages 69-85
  7. Grazyna Kochan, Holly Stephenson, Karine Breckpot, David Escors
    Pages 87-104

About this book


Gene therapy was conceived during the early and mid part of the 20th century. At first, it was considered a revolutionary biomedical procedure, which could potentially cure any disease for which the molecular bases were understood. Since then, gene therapy has gone through many stages and has evolved from a nearly unrealistic perspective to a real life application. Clinical efficacy in humans was demonstrated at the beginning of this century after its successful application in small-scale clinical trials to cure severe immunodeficiency in children. However, their successes were overshadowed some time later by the occurrence of vector-related leukaemia in a number of treated children. It is in this context that lentiviral vectors have appeared, with improved efficiency and, possibly, increased biosafety. Very recently, the first clinical trials with lentivectors have been carried out with some success.

This Brief firstly defines gene therapy, and places lentivectors within this fascinating therapeutic strategy. Then follows a comprehensive description of the development of retroviral and lentiviral vectors and how to specifically target distinct cell types and tissues. The authors also discuss the application of lentivector gene therapy for the treatment of cancer and autoimmune diseases, ending with the application of lentivectors in human gene therapy clinical trials.


cancer clinical trial gene therapy lentivector promoter viral immunology

Authors and affiliations

  • David Escors
    • 1
  • Karine Breckpot
    • 2
  • Frederick Arce
    • 3
  • Grazyna Kochan
    • 4
  • Holly Stephenson
    • 5
  1. 1.Rayne Institute, Division of Infection and ImmunityUniversity CollegeLondonUnited Kingdom
  2. 2.Medical School, Department of Physiology-ImmunologyFree University of BrusselsJetteBelgium
  3. 3.University College LondonLondonUnited Kingdom
  4. 4., Nuffield Orthopaedic CentreUniversity of OxfordOxfordUnited Kingdom
  5. 5.University College LondonLondonUnited Kingdom

Bibliographic information