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Blood, 1 May 2008, Vol. 111, No. 9, pp. 4431-4444. This Article Full Text Full Text (PDF) Alert me when this article is cited Alert me if a correction is posted Citation Map 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 Nienhuis, A. W. Search for Related Content PubMed PubMed Citation Articles by Nienhuis, A. W. Related Collections Free Research Articles ASH 50th Anniversary Reviews Gene Therapy Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  ASH 50TH ANNIVERSARY REVIEW Development of gene therapy for blood disorders Arthur W. Nienhuis1 1 Division of Experimental Hematology, Department of Hematology, St Jude Children's Research Hospital, Memphis, TN The concept of introducing genes into human cells for therapeutic purposes developed nearly 50 years ago as diseases due to defects in specific genes were recognized. Development of recombinant DNA techniques in the 1970s and their application to the study of mouse tumor viruses facilitated the assembly of the first gene transfer vectors. Vectors of several different types have now been developed for specific applications and over the past decade, efficacy has been demonstrated in many animal models. Clinical trials began in 1989 and by 2002 there was unequivocal evidence that children with severe combined immunodeficiency could be cured by gene transfer into primitive hematopoietic cells. Emerging from these successful trials was the realization that proto-oncogene activation by retroviral integration could contribute to leukemia. Much current effort is focused on development of safer vectors. Successful gene therapy applications have also been developed for control of graft-versus-host disease and treatment of various viral infections, leukemias, and lymphomas. The hemophilias seem amenable to gene therapy intervention and informative clinical trials have been conducted. The hemoglobin disorders, an early target for gene therapy, have proved particularly challenging although ongoing research is yielding new information that may ultimately lead to successful clinical trials. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: N. Uchida, K. N. Washington, J. Hayakawa, M. M. Hsieh, A. C. Bonifacino, A. E. Krouse, M. E. Metzger, R. E. Donahue, and J. F. Tisdale Development of a Human Immunodeficiency Virus Type 1-Based Lentiviral Vector That Allows Efficient Transduction of both Human and Rhesus Blood Cells J. Virol., October 1, 2009; 83(19): 9854 - 9862. [Abstract] [Full Text] [PDF] H. Zhao, T. I. Pestina, M. Nasimuzzaman, P. Mehta, P. W. Hargrove, and D. A. Persons Amelioration of murine {beta}-thalassemia through drug selection of hematopoietic stem cells transduced with a lentiviral vector encoding both {gamma}-globin and the MGMT drug-resistance gene Blood, June 4, 2009; 113(23): 5747 - 5756. [Abstract] [Full Text] [PDF] A. N. Schechter Hemoglobin research and the origins of molecular medicine Blood, November 15, 2008; 112(10): 3927 - 3938. [Abstract] [Full Text] [PDF]

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