Gene therapy for adenosine deaminase deficiency

Barbara Cappelli, Alessandro Aiuti

Research output: Contribution to journalArticlepeer-review

Abstract

In the last decade, gene therapy for adenosine deaminase deficiency has been developed as a successful alternative strategy to allogeneic bone marrow transplant and enzyme replacement therapy. Infusion of autologous hematopoietic stem cells, corrected ex vivo by retroviral vectors and combined to low-intensity conditioning regimen, has resulted in immunologic improvement, metabolic correction, and long-term clinical benefits. These findings have opened the way to applications of gene therapy in other primary immune deficiencies using novel vector technology.

Original languageEnglish
Pages (from-to)249-260
Number of pages12
JournalImmunology and Allergy Clinics of North America
Volume30
Issue number2
DOIs
Publication statusPublished - 2010

Keywords

  • Adenosine deaminase
  • Gene therapy
  • Haematopoietic stem cell
  • Immune deficiency
  • Reduced-intensity conditioning regimen

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology
  • Medicine(all)

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