Gene therapy for primary immunodeficiencies: Part 1

Marina Cavazzana-Calvo, Alain Fischer, Salima Hacein-Bey-Abina, Alessandro Aiuti

Research output: Contribution to journalArticlepeer-review


Over 60 patients affected by SCID due to IL2RG deficiency (SCID-X1) or adenosine deaminase (ADA)-SCID have received hematopoietic stem cell gene therapy in the past 15 years using gammaretroviral vectors, resulting in immune reconstitution and clinical benefit in the majority of them. However, the occurrence of insertional oncogenesis in the SCID-X1 trials has led to the development of new clinical trials based on integrating vectors with improved safety design as well as investigation on new technologies for highly efficient gene targeting and site-specific gene editing. Here we will present the experience and perspectives of gene therapy for SCID-X1 and ADA-SCID and discuss the pros and cons of gene therapy in comparison to allogeneic transplantation.

Original languageEnglish
Pages (from-to)580-584
Number of pages5
JournalCurrent Opinion in Immunology
Issue number5
Publication statusPublished - Oct 2012

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology


Dive into the research topics of 'Gene therapy for primary immunodeficiencies: Part 1'. Together they form a unique fingerprint.

Cite this