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Prospects for Gene Therapy for Immunodeficiency Diseases

Prospects for Gene Therapy for Immunodeficiency Diseases The fields of gene transfer and expression have recently seen major advances that have made possible a better understanding of basic biologic mechanisms and have brought closer the possibility for gene therapy for the treatment of clinical disease. Vectors derived from murine retroviruses are being developed for use in clinical protocols. In this article we address the recent advances in gene transfer as they relate to progress toward achieving gene therapy for immunodeficiency diseases, specifically for adenosine deaminase (ADA) deficiency as a cause of severe combined immunodeficiency (SCID). It now appears that ADA deficiency will be the first disease in which the technique of retroviral-mediated gene therapy will be attempted. METHODS OF GENE TRANSFER A number of techniques have been used successfully to introduce cloned genes into mammalian cells in culture (for a detailed review, see Ref. 1). The major methods can be grouped in four categories: (a) viral, both RNA viruses (e.g. retroviruses) and DNA viruses (e.g. SV40, adenovirus, and I The US Government has the right to retain a nonexclusive royalty-free license in and to any copyright covering this paper. KANTOFF, FREEMAN & ANDERSON bovine papilloma); (b) chemical, such as calcium phosphate-mediated DNA uptake; (c) physical, http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Annual Review of Immunology Annual Reviews

Prospects for Gene Therapy for Immunodeficiency Diseases

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Publisher
Annual Reviews
Copyright
Copyright 1988 Annual Reviews. All rights reserved
Subject
Review Articles
ISSN
0732-0582
eISSN
1545-3278
DOI
10.1146/annurev.iy.06.040188.003053
pmid
3289578
Publisher site
See Article on Publisher Site

Abstract

The fields of gene transfer and expression have recently seen major advances that have made possible a better understanding of basic biologic mechanisms and have brought closer the possibility for gene therapy for the treatment of clinical disease. Vectors derived from murine retroviruses are being developed for use in clinical protocols. In this article we address the recent advances in gene transfer as they relate to progress toward achieving gene therapy for immunodeficiency diseases, specifically for adenosine deaminase (ADA) deficiency as a cause of severe combined immunodeficiency (SCID). It now appears that ADA deficiency will be the first disease in which the technique of retroviral-mediated gene therapy will be attempted. METHODS OF GENE TRANSFER A number of techniques have been used successfully to introduce cloned genes into mammalian cells in culture (for a detailed review, see Ref. 1). The major methods can be grouped in four categories: (a) viral, both RNA viruses (e.g. retroviruses) and DNA viruses (e.g. SV40, adenovirus, and I The US Government has the right to retain a nonexclusive royalty-free license in and to any copyright covering this paper. KANTOFF, FREEMAN & ANDERSON bovine papilloma); (b) chemical, such as calcium phosphate-mediated DNA uptake; (c) physical,

Journal

Annual Review of Immunology – Annual Reviews

Published: Apr 1, 1988

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