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Prepublished online as a Blood First Edition Paper on January 2, 2003; DOI 10.1182/blood-2002-07-2314. This Article Full Text (PDF) All Versions of this Article: 2002-07-2314v1 101/6/2099    most recent Alert me when this article is cited Alert me if a correction is posted Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Baum, C. Articles by von Kalle, C. Search for Related Content PubMed PubMed Citation Articles by Baum, C. Articles by von Kalle, C. Social Bookmarking                   What's this? Submitted July 31, 2002 Accepted December 26, 2002 Side effects of retroviral gene transfer into hematopoietic stem cells Christopher Baum*, Jochen Duellmann, Zhixiong Li, Boris Fehse, Johann Meyer, David A Williams, and Christof von Kalle Department of Hematology & Oncology, Hannover Medical School, Hannover, Germany Department of Cell & Virus Genetics, Heinrich Pette Institute, Hamburg, Germany Department of Neuroanatomy, University Hospital Eppendorf, Hamburg, Germany Department of Bone Marrow Transplantation, University Hospital Eppendorf, Hamburg, Germany Experimental Hematology, Cincinnati Children's Research Foundation, Cincinnati, OH, USA * Corresponding author; email: baum.christopher{at}mh-hannover.de. Recent conceptual and technical improvements have resulted in clinically meaningful levels of gene transfer into repopulating hematopoietic stem cells. At the same time, evidence is accumulating that gene therapy may induce several kinds of unexpected side effects, based on preclinical and clinical data. To assess the therapeutic potential of genetic interventions in hematopoietic cells, it will be important to derive a classification of side effects, to obtain insights into their underlying mechanisms and to use rigorous statistical approaches in comparing data. We here review side effects related to target cell manipulation, vector production, transgene insertion and expression, selection procedures for transgenic cells, and immune surveillance. We also address some inherent differences between hematopoiesis in the most commonly used animal model, the laboratory mouse, and in humans. It is our intention to emphasize the need for a critical and hypothesis-driven analysis of 'transgene toxicology', in order to improve safety, efficiency and prognosis for the yet small but expanding group of patients that could benefit from gene therapy. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: U. Baranyi, B. Linhart, N. Pilat, M. Gattringer, J. Bagley, F. Muehlbacher, J. Iacomini, R. Valenta, and T. Wekerle Tolerization of a Type I Allergic Immune Response through Transplantation of Genetically Modified Hematopoietic Stem Cells J. Immunol., June 15, 2008; 180(12): 8168 - 8175. [Abstract] [Full Text] [PDF] A. W. Nienhuis Development of gene therapy for blood disorders Blood, May 1, 2008; 111(9): 4431 - 4444. [Abstract] [Full Text] [PDF] U. Blank, G. Karlsson, and S. Karlsson Signaling pathways governing stem-cell fate Blood, January 15, 2008; 111(2): 492 - 503. [Abstract] [Full Text] [PDF] L. 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