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|a 10.1016/j.clim.2022.109084
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|a eng
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1 |
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|a Slatter, Mary A
|e verfasserin
|4 aut
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|a Advances in the treatment of severe combined immunodeficiency
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|c 2022
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|a Text
|b txt
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|a ƒaComputermedien
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|a Date Completed 13.09.2022
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|a Date Revised 11.11.2022
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|a published: Print-Electronic
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|a Citation Status MEDLINE
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|a Copyright © 2022 Elsevier Inc. All rights reserved.
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|a Severe Combined Immunodeficiency (SCID) is the most profound inborn error of immunity affecting cellular and humoral immunity. Hematopoietic stem cell transplantation has been a curative treatment since 1968. Huge progress has been made since then in understanding the underlying genetics, improving outcomes from transplant, and introducing gene therapy in particular for adenosine deaminase deficient- and IL2 receptor gamma-deficient SCID. Newborn screening has been widely introduced across the world to enable definitive treatment before infection occurs. This article aims to review the latest evidence on how to achieve curative treatment with minimal short- and long-term toxicity, normal immune reconstitution and good quality of life
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|a Journal Article
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|a Review
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|a Gene therapy
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|a Hematopoioetic stem cell transplantation
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|a Newborn screening
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| 650 |
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|a Severe combined immunodeficiency
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| 650 |
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|a Adenosine Deaminase
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|a Gennery, Andrew R
|e verfasserin
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|i Enthalten in
|t Clinical immunology (Orlando, Fla.)
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