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Gene Therapy of the Immune System

Gene Therapy of the Immune System Many applications of somatic gene therapy relate to the immune system. Several forms ofinherited immunodeficiencies are candidates for treatment by gene transfer. Adenosine deaminase (ADA) deficiency causes a form of severe combined immunodeficiency. Stable gene transfer and expression of human ADA has now been obtained in hematopoietic stem cells of mice and, more recently, in large animals. The human ADA has also been introduced and expressed in the primitive human hematopoietic progenitor cells that initiate long-term bone marrow culture. Clinical trials of gene therapy for ADA deficiency have been initiated. The initial protocols were aimed at the correction of peripheral blood T lymphocytes, but recent strategies are attempting ADA gene transfer into peripheral blood or bone marrow stem cells. Other immunodeficiencies that may soon be amenable to somatic gene therapy include leukocyte adhesion deficiency and chronic granulomatous disease. Gene therapy may also be applied to the treatment of acquired disorders. In theory, the hematopoietic stem cells of a human immunodeficiency virus (HIV)-infected patient could be genetically modi­ fied and used to reconstitute an HIV-resistant hematopoietic system. Vari­ ous strategies are currently being investigated to achieve this "intracellular immunization" against HIV. These include the transfer of genes encoding 297 http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Annual Review of Immunology Annual Reviews

Gene Therapy of the Immune System

Cournoyer, D; Caskey, C T
Annual Review of Immunology , Volume 11 (1) – Apr 1, 1993
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Publisher
Annual Reviews
Copyright
Copyright 1993 Annual Reviews. All rights reserved
Subject
Review Articles
ISSN
0732-0582
eISSN
1545-3278
DOI
10.1146/annurev.iy.11.040193.001501
pmid
8476564
Publisher site
See Article on Publisher Site

Abstract

Many applications of somatic gene therapy relate to the immune system. Several forms ofinherited immunodeficiencies are candidates for treatment by gene transfer. Adenosine deaminase (ADA) deficiency causes a form of severe combined immunodeficiency. Stable gene transfer and expression of human ADA has now been obtained in hematopoietic stem cells of mice and, more recently, in large animals. The human ADA has also been introduced and expressed in the primitive human hematopoietic progenitor cells that initiate long-term bone marrow culture. Clinical trials of gene therapy for ADA deficiency have been initiated. The initial protocols were aimed at the correction of peripheral blood T lymphocytes, but recent strategies are attempting ADA gene transfer into peripheral blood or bone marrow stem cells. Other immunodeficiencies that may soon be amenable to somatic gene therapy include leukocyte adhesion deficiency and chronic granulomatous disease. Gene therapy may also be applied to the treatment of acquired disorders. In theory, the hematopoietic stem cells of a human immunodeficiency virus (HIV)-infected patient could be genetically modi­ fied and used to reconstitute an HIV-resistant hematopoietic system. Vari­ ous strategies are currently being investigated to achieve this "intracellular immunization" against HIV. These include the transfer of genes encoding 297

Journal

Annual Review of Immunology – Annual Reviews

Published: Apr 1, 1993

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