Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID

Alessandro Aiuti, Immacolata Brigida, Francesca Ferrua, Barbara Cappelli, Robert Chiesa, Sarah Marktel, Maria Grazia Roncarolo

Research output: Contribution to journalArticlepeer-review


Gene therapy is a highly attractive strategy for many types of inherited disorders of the immune system. Adenosine deaminase (ADA) deficient-severe combined immunodeficiency (SCID) has been the target of several clinical trials based on the use of hematopoietic stem/progenitor cells engineered with retroviral vectors. The introduction of a low intensity conditioning regimen has been a crucial factor in achieving stable engrafment of hematopoietic stem cells and therapeutic levels of ADA-expressing cells. Recent studies have demonstrated that gene therapy for ADA-SCID has favorable safety profile and is effective in restoring normal purine metabolism and immune functions. Stem cell gene therapy combined with appropriate conditioning regimens might be extended to other genetic disorders of the hematopoietic system.

Original languageEnglish
Pages (from-to)150-159
Number of pages10
JournalImmunologic Research
Issue number1-3
Publication statusPublished - 2009


  • Bone marrow
  • Gene therapy
  • Hematopoietic stem cell
  • Retroviral vector
  • Severe combined immunodeficiency

ASJC Scopus subject areas

  • Immunology


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