Abstract
Gene therapy has become an attractive alternative therapeutic strategy to allogeneic transplant for primary immunodeficiencies (PIDs) owing to known genetic defects. Clinical trials using gammaretroviral vectors have demonstrated the proof of principle of gene therapy for Wiskott-Aldrich syndrome (WAS) and chronic granulomatous disease (CGD), but have also highlighted limitations of the technology. New strategies based on vectors that can achieve more robust correction with less risk of insertional mutagenesis are being developed. In this review we present the status of gene therapy for WAS and CGD, and discuss the emerging application of similar strategies to a broader range of PIDs, such as IPEX syndrome.
Copyright © 2012. Published by Elsevier Ltd.
Publication types
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Research Support, Non-U.S. Gov't
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Review
MeSH terms
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Animals
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Clinical Trials as Topic / trends
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Exfoliation Syndrome / genetics
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Exfoliation Syndrome / immunology
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Exfoliation Syndrome / therapy
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Forecasting / methods
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Gene Transfer Techniques / trends
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Genetic Therapy / methods*
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Genetic Therapy / trends*
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Genetic Vectors / genetics
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Genetic Vectors / immunology
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Genetic Vectors / therapeutic use
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Granulomatous Disease, Chronic / genetics
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Granulomatous Disease, Chronic / immunology
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Granulomatous Disease, Chronic / therapy
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Humans
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Immunologic Deficiency Syndromes / genetics*
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Immunologic Deficiency Syndromes / immunology
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Immunologic Deficiency Syndromes / therapy*
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Severe Combined Immunodeficiency / genetics
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Severe Combined Immunodeficiency / immunology
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Severe Combined Immunodeficiency / therapy
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Wiskott-Aldrich Syndrome / genetics
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Wiskott-Aldrich Syndrome / immunology
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Wiskott-Aldrich Syndrome / therapy