Current Status of Retroviral Vector Mediated Gene Transfer into Human Hematopoietic Stem Cells

Genetic modification of hematopoietic stem cells (HSCs) has been proposed as a treatment strategy for a variety of hematologic diseases, tracking marked cells or conferring resistance to chemotherapeutic agents. Despite early enthusiasm, the results of clinical studies involving gene transfer into H...

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Bibliographic Details
Published in:Leukemia & lymphoma 2001, Vol.41 (1-2), p.1-18
Main Authors: Elwood, Ngaire J., Smith, Clay A.
Format: Article
Language:English
Subjects:
Animals
gene therapy
Genetic Vectors - standards
Genetic Vectors - therapeutic use
Hematologic Diseases - therapy
hematopoietic stem cells
Hematopoietic Stem Cells - cytology
Hematopoietic Stem Cells - metabolism
Humans
Retroviridae - genetics
Transduction, Genetic - methods
Transduction, Genetic - standards
vectors
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Summary:Genetic modification of hematopoietic stem cells (HSCs) has been proposed as a treatment strategy for a variety of hematologic diseases, tracking marked cells or conferring resistance to chemotherapeutic agents. Despite early enthusiasm, the results of clinical studies involving gene transfer into HSCs have not resulted in therapeutic benefits for the vast majority of treated patients. This review describes the limitations and advances that have been made in the areas of gene transfer vectors, identification of the appropriate HSCs to target for genetic modifications and the methods used to perform gene transfer.
ISSN:1042-8194
1029-2403
DOI:10.3109/10428190109057950