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Gene therapy for primary immunodeficiencies: part 2 - Zurich Open Repository and Archive


Aiuti, Alessandro; Bacchetta, Rosa; Seger, Reinhard; Villa, Anna; Cavazzana-Calvo, Marina (2012). Gene therapy for primary immunodeficiencies: part 2. Current Opinion in Immunology, 24(5):585-591.

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.

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.

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36 citations in Web of Science®
38 citations in Scopus®
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Additional indexing

Item Type:Journal Article, refereed, original work
Communities & Collections:04 Faculty of Medicine > University Children's Hospital Zurich > Medical Clinic
Dewey Decimal Classification:610 Medicine & health
Language:English
Date:2012
Deposited On:24 Jan 2013 14:27
Last Modified:05 Apr 2016 16:19
Publisher:Elsevier
ISSN:0952-7915
Publisher DOI:https://doi.org/10.1016/j.coi.2012.07.012
PubMed ID:22909900

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