Biosafety in Handling Gene Transfer Vectors

Andrew Braun1

1 Harvard Medical School, Boston, Massachusetts
Publication Name:  Current Protocols in Human Genetics
Unit Number:  Unit 12.1
DOI:  10.1002/0471142905.hg1201s50
Online Posting Date:  August, 2006
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This unit is devoted to safety issues that must be considered when generating and working with vectors for gene therapy. A short description of the relevant regulatory bodies and mechanisms is followed by a general discussion of containment and safety procedures. This is followed by vector‐specific guidelines for nine commonly used vector systems, including adenovirus, vaccinia, HSV‐1, lentivirus, and nonviral vectors. Recent tragic experiences with retroviral integration near oncogene loci demonstrate the surprise effects of successful gene therapy. The unit also outlines the problems encountered when taking a promising vector to clinical trials.

Keywords: biosafety; gene transfer; vectors; hazards; risks

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Table of Contents

  • Introduction
  • Background
  • Running A BL‐2 Lab
  • Specific Vectors
  • Getting a Useful Vector into Clinical Trials
  • Further Reading
  • Literature Cited
  • Figures
  • Tables
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Literature Cited

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Internet Resources
  Biosafety in Microbiological and Biomedical Laboratories (BMBL), from the Centers for Disease Control (CDC) Office of Health Safety (OHS) and the NIH.
  FDA Center for Biologics Evaluation and Research (CBER) home page.
  FDA guidelines for human somatic cell therapy and gene therapy.
  Guidance for Testing Replication Competent Retrovirus, FDA, October 2000.
  NIH Office of Biotechnology Activities (OBA) home page, providing a wide range of guidelines and other useful documents.
  APPENDIX M of the April 2002 NIH Recombinant DNA guidelines, listing recombinant DNA agents by risk groups.
  APPENDIX M of the April 2002 NIH Recombinant DNA Guidelines, consisting of a series of screening questions concerning potential vectors and their application to human gene therapy.
  List of human gene transfer studies registered with the NIH.
  NIH Office of Research Facilities' Research Laboratory Design Policy and Guidelines.
  Biosafety Manual from the University of Pennsylvania
  Biosafety Manual from Stanford University
  Biosafety Manual from Princeton University (contains a particularly useful index)
  Home page of the National Gene Vector Laboratories (NGVL), a group set up under NIH auspices to help researchers obtain clinical‐grade human gene transfer vectors.
  Announcement of 3rd SAE in Necker University Hospital X‐SCID series.
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