Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

Deficiency of adenosine deaminase (ADA) causes a form of severe combined immunodeficiency disease. This study shows that gene therapy in the form of hematopoietic stem cells transduced with a viral vector containing the human ADA gene can restore immune function in patients with this disease. Defici...

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Veröffentlicht in:	The New England journal of medicine 2009-01, Vol.360 (5), p.447-458
Hauptverfasser:	Aiuti, Alessandro, Cattaneo, Federica, Galimberti, Stefania, Benninghoff, Ulrike, Cassani, Barbara, Callegaro, Luciano, Scaramuzza, Samantha, Andolfi, Grazia, Mirolo, Massimiliano, Brigida, Immacolata, Tabucchi, Antonella, Carlucci, Filippo, Eibl, Martha, Aker, Memet, Slavin, Shimon, Al-Mousa, Hamoud, Al Ghonaium, Abdulaziz, Ferster, Alina, Duppenthaler, Andrea, Notarangelo, Luigi, Wintergerst, Uwe, Buckley, Rebecca H, Bregni, Marco, Marktel, Sarah, Valsecchi, Maria Grazia, Rossi, Paolo, Ciceri, Fabio, Miniero, Roberto, Bordignon, Claudio, Roncarolo, Maria-Grazia
Format:	Artikel
Sprache:	eng
Schlagworte:	Adenosine Deaminase - deficiency Adenosine Deaminase - genetics Antigens, CD34 - genetics Biological and medical sciences Bone marrow Bone Marrow Cells - immunology Child, Preschool Combined Modality Therapy Ethics Follow-Up Studies Gene therapy General aspects Genetic Therapy Genetic Vectors Hematopoietic Stem Cell Transplantation Humans Immunodeficiencies Immunodeficiencies. Immunoglobulinopathies Immunopathology Infant Kinases Ligands Lymphocyte Count Medical sciences Metabolites Patients Retroviridae Severe Combined Immunodeficiency - immunology Severe Combined Immunodeficiency - therapy Transduction, Genetic Transplantation Conditioning Transplants & implants
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