Autoimmune dysregulation and purine metabolism in adenosine deaminase deficiency. 2012

Aisha Vanessa Sauer, and Immacolata Brigida, and Nicola Carriglio, and Alessandro Aiuti
San Raffaele Telethon Institute for Gene Therapy Milan, Italy.

Genetic defects in the adenosine deaminase (ADA) gene are among the most common causes for severe combined immunodeficiency (SCID). ADA-SCID patients suffer from lymphopenia, severely impaired cellular and humoral immunity, failure to thrive, and recurrent infections. Currently available therapeutic options for this otherwise fatal disorder include bone marrow transplantation (BMT), enzyme replacement therapy with bovine ADA (PEG-ADA), or hematopoietic stem cell gene therapy (HSC-GT). Although varying degrees of immune reconstitution can be achieved by these treatments, breakdown of tolerance is a major concern in ADA-SCID. Immune dysregulation such as autoimmune hypothyroidism, diabetes mellitus, hemolytic anemia, and immune thrombocytopenia are frequently observed in milder forms of the disease. However, several reports document similar complications also in patients on long-term PEG-ADA and after BMT or GT treatment. A skewed repertoire and decreased immune functions have been implicated in autoimmunity observed in certain B-cell and/or T-cell immunodeficiencies, but it remains unclear to what extent specific mechanisms of tolerance are affected in ADA deficiency. Herein we provide an overview about ADA-SCID and the autoimmune manifestations reported in these patients before and after treatment. We also assess the value of the ADA-deficient mouse model as a useful tool to study both immune and metabolic disease mechanisms. With focus on regulatory T- and B-cells we discuss the lymphocyte subpopulations particularly prone to contribute to the loss of self-tolerance and onset of autoimmunity in ADA deficiency. Moreover we address which aspects of immune dysregulation are specifically related to alterations in purine metabolism caused by the lack of ADA and the subsequent accumulation of metabolites with immunomodulatory properties.

UI MeSH Term Description Entries

Related Publications

Aisha Vanessa Sauer, and Immacolata Brigida, and Nicola Carriglio, and Alessandro Aiuti
January 1978, Ciba Foundation symposium,
Aisha Vanessa Sauer, and Immacolata Brigida, and Nicola Carriglio, and Alessandro Aiuti
August 1976, Proceedings of the National Academy of Sciences of the United States of America,
Aisha Vanessa Sauer, and Immacolata Brigida, and Nicola Carriglio, and Alessandro Aiuti
January 1985, Annals of the New York Academy of Sciences,
Aisha Vanessa Sauer, and Immacolata Brigida, and Nicola Carriglio, and Alessandro Aiuti
May 1978, Clinical science and molecular medicine,
Aisha Vanessa Sauer, and Immacolata Brigida, and Nicola Carriglio, and Alessandro Aiuti
July 1990, Seminars in hematology,
Aisha Vanessa Sauer, and Immacolata Brigida, and Nicola Carriglio, and Alessandro Aiuti
May 1998, Clinical and diagnostic laboratory immunology,
Aisha Vanessa Sauer, and Immacolata Brigida, and Nicola Carriglio, and Alessandro Aiuti
October 1978, Clinical and experimental immunology,
Aisha Vanessa Sauer, and Immacolata Brigida, and Nicola Carriglio, and Alessandro Aiuti
August 1977, Nihon rinsho. Japanese journal of clinical medicine,
Aisha Vanessa Sauer, and Immacolata Brigida, and Nicola Carriglio, and Alessandro Aiuti
July 1976, Pediatric research,
Aisha Vanessa Sauer, and Immacolata Brigida, and Nicola Carriglio, and Alessandro Aiuti
June 2011, The Journal of allergy and clinical immunology,
Copied contents to your clipboard!