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

@article{Ferrua2010UpdateOG,
  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},
  year={2010},
  volume={10},
  pages={551–556}
}
Purpose of reviewThe 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 findingsSince 2000, more than 30 ADA-SCID patients have been treated with gene therapy worldwide, with successful outcome in most cases, consisting… Expand
Gene therapy for primary adaptive immune deficiencies.
TLDR
Gene therapy has become an option for the treatment of 2 forms of severe combined immunodeficiency (SCID): X-linked SCID and adenosine deaminase deficiency and its safe and effective extension will have to be proved by examining the results of the ongoing clinical trials. Expand
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TLDR
This review will outline the impact of ADA deficiency on various organ systems, starting with the well-understood immunological abnormalities and suggest that it should be recognized as a systemic metabolic disorder that affects multiple organ systems. Expand
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TLDR
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TLDR
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