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.
journal_name
Curr Opin Immunoljournal_title
Current opinion in immunologyauthors
Aiuti A,Bacchetta R,Seger R,Villa A,Cavazzana-Calvo Mdoi
10.1016/j.coi.2012.07.012subject
Has Abstractpub_date
2012-10-01 00:00:00pages
585-91issue
5eissn
0952-7915issn
1879-0372pii
S0952-7915(12)00121-5journal_volume
24pub_type
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