Progress in gene therapy for primary immunodeficiencies using lentiviral vectors

Aisha V. Sauer, Biagio Di Lorenzo, Nicola Carriglio, Alessandro Aiuti

Research output: Contribution to journalArticlepeer-review


Gene therapy for PIDs is an effective treatment, providing potential long-term clinical benefit for affected patients. Substantial progress has been made to make lentiviral gene therapy platforms available for a number of rare genetic diseases. Although many ongoing gene therapy trials are based on ex-vivo approaches with autologous hematopoietic stem cells, other approaches such as in-vivo gene therapy or gene-repair platforms might provide further advancement for certain PIDs.

Original languageEnglish
Pages (from-to)527-534
Number of pages8
JournalCurrent Opinion in Allergy and Clinical Immunology
Issue number6
Publication statusPublished - 2014


  • Gene therapy
  • Lentiviral vector
  • Primary immunodeficiency
  • Severe combined immunodeficiency

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology


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