Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency.

  title={Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency.},
  author={Francesca Ferrua and Immacolata Brigida and Alessandro Aiuti},
  journal={Current opinion in allergy and clinical immunology},
  volume={10 6},
PURPOSE OF REVIEW The present review describes the recent progress in the treatment of adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID) using autologous gene-modified hematopoietic stem cells, comparing immune reconstitution with respect to allogeneic transplant and discussing differences with gene therapy for SCID-X1. RECENT FINDINGS Since 2000, more than 30 ADA-SCID patients have been treated with gene therapy worldwide, with successful outcome in most cases… CONTINUE READING
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Gene therapy for adenosine deaminase deficiency.

Immunology and allergy clinics of North America • 2010

Efficacy of gene therapy for X - linked severe combined immunodeficiency

B Cassani, M Mirolo, F Cattaneo
Hematology Am Soc Hematol Educ Program • 2009

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