27. Toxicology for Gene Therapy Products: Concepts and Challenges

  1. Boro Dropulic2 and
  2. Barrie J. Carter3
  1. Russette M. Lyons

Published Online: 11 APR 2007

DOI: 10.1002/9780470184585.ch27

Concepts in Genetic Medicine

Concepts in Genetic Medicine

How to Cite

Lyons, R. M. (2008) Toxicology for Gene Therapy Products: Concepts and Challenges, in Concepts in Genetic Medicine (eds B. Dropulic and B. J. Carter), John Wiley & Sons, Inc., Hoboken, NJ, USA. doi: 10.1002/9780470184585.ch27

Editor Information

  1. 2

    Lentigen Corporation, Baltimore, Maryland, USA

  2. 3

    Targeted Genetics Corporation, Seattle, Washington, USA

Author Information

  1. Novartis Vaccines and Diagnostics, Cambridge, Massachusetts, USA

Publication History

  1. Published Online: 11 APR 2007
  2. Published Print: 22 JAN 2008

ISBN Information

Print ISBN: 9780471703204

Online ISBN: 9780470184585



  • small-molecule toxicology plans;
  • no observable adverse effect level (NOAEL);
  • chronic and progressive toxicological responses


This chapter contains sections titled:

  • Fundamental Toxicological Concepts

  • Dose Relationship

  • Exposure

  • Types of Adverse Effects

  • Toxicology Study Objectives

  • Toxicology Challenges for Gene Therapy Products

  • References