Abstract
Prior to the first successful bone marrow transplant in 1968, patients born with severe combined immunodeficiency (SCID) invariably died. Today, with a widening availability of newborn screening, major improvements in the application of allogeneic procedures, and the emergence of successful hematopoietic stem and progenitor cell (HSC/P) gene therapy, the majority of these children can be identified and cured. Here, we trace key steps in the development of clinical gene therapy for SCID and other primary immunodeficiencies (PIDs), and review the prospects for adoption of new targets and technologies.
Keywords:
gene therapy; immunodeficiencies; retrovirus.
Copyright © 2017 The American Society of Gene and Cell Therapy. Published by Elsevier Inc. All rights reserved.
Publication types
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Historical Article
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Review
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Research Support, Non-U.S. Gov't
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Research Support, N.I.H., Extramural
MeSH terms
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Adenosine Deaminase / deficiency
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Adenosine Deaminase / genetics*
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Adenosine Deaminase / immunology
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Clinical Trials as Topic
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Gammaretrovirus / genetics
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Gammaretrovirus / immunology
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Gene Expression
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Genetic Therapy / history
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Genetic Therapy / methods*
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Genetic Vectors / chemistry
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Genetic Vectors / immunology
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Hematopoietic Stem Cell Transplantation / methods*
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History, 20th Century
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History, 21st Century
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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 / pathology
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Immunologic Deficiency Syndromes / therapy*
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Lentivirus / genetics
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Lentivirus / immunology
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Severe Combined Immunodeficiency / genetics
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Severe Combined Immunodeficiency / immunology
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Severe Combined Immunodeficiency / pathology
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Severe Combined Immunodeficiency / therapy*
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Transplantation, Homologous