Genetic Engineering Strategies for Hematologic Malignancies

  • Thomas J. Kipps
Part of the Genetic Engineering book series (GEPM, volume 22)


Hematologic malignancies have several features that make them particularly well suited for treatment with the use of genetic engineering approaches. First, genetic engineering of stem cells can enhance the resistance of marrow progenitor cells to chemotherapy, potentially enhancing the therapeutic index of conventional therapy. Second, non-neoplastic cells that infiltrate the leukemia population also are present in the blood, making these cells accessible for isolation, analysis, genetic manipulation and subsequent use as effector cells in adoptive immune therapy. Third, because the neoplastic cells circulate in blood, large numbers of tumor cells can be harvested for genetic manipulation ex vivo. In addition, relatively simple blood tests can be used to monitor for expression of a selected transgene by neoplastic cells in vivo or for induced phenotypic changes on leukemia and bystander cells following gene transfer. Finally, recent advances in molecular biology and immunology have identified features of hematologic malignancies that potentially could be corrected through gene transfer for therapeutic benefit. As such, hematologic malignancies are highly amenable for development of novel gene therapy approaches.


Leukemia Cell Hematologic Malignancy Thymidine Kinase Acute Myelogenous Leukemia Mixed Lymphocyte Reaction 
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© Springer Science+Business Media New York 2000

Authors and Affiliations

  • Thomas J. Kipps
    • 1
  1. 1.San Diego Division of Hematology and Oncology Human Gene Therapy Program Department of MedicineUniversity of CaliforniaLa JollaUSA

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