Genetic modification of the hematolymphoid compartment for therapeutic purposes.
Gene transfer into cells of the hematolymphoid compartment has enormous potential to provide effective therapies across a wide spectrum of human disease. This article reviews current and potential uses of gene modification in this compartment. Issues relevant to the eventual success of gene therapy in the hematolymphoid compartment are also discussed, including biological features of target cells, ex vivo versus in vivo gene therapy approaches, and currently available vector technologies.
Department of Molecular and Cellular Engineering, University of Pennsylvania, Philadelphia, USA. kerrw@mail.med.upenn.edu
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D000163:Acquired Immunodeficiency Syndrome / Q000628:therapy; D030342:Genetic Diseases, Inborn / Q000628:therapy; D015316:Genetic Therapy / Q000379:methods*; D006412:Hematopoietic Stem Cells / Q000378:metabolism*; D006801:Humans; D008208:Lymphatic System / Q000378:metabolism*; D009369:Neoplasms / Q000628:therapy; D013601:T-Lymphocytes / Q000378:metabolism*