Journal
CURRENT OPINION IN IMMUNOLOGY
Volume 24, Issue 5, Pages 585-591Publisher
CURRENT BIOLOGY LTD
DOI: 10.1016/j.coi.2012.07.012
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Funding
- European Commission: Advanced Cell-based Therapies for the treatment of Primary ImmunoDeficiency (Cell-PID) [HEALTH F5-2010-261387]
- Italian Telethon Foundation
- Fondazione Roma
- Italian Ministry of Research and University (MIUR)
- European Research Council (ERC)
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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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