Gene therapy for primary immunodeficiencies: Part 2

Alessandro Aiuti, Rosa Bacchetta, Reinhard Seger, Anna Villa, Marina Cavazzana-Calvo

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish
Pages (from-to)585-591
Number of pages7
JournalCurrent Opinion in Immunology
Issue number5
Publication statusPublished - Oct 2012

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology


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