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Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

โœ Scribed by 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


Book ID
120321930
Publisher
Massachusetts Medical Society
Year
2009
Tongue
English
Weight
535 KB
Volume
360
Category
Article
ISSN
0096-6762

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Severe combined immunodeficiency disease
โœ Robertson Parkman; Erwin W Gelfand ๐Ÿ“‚ Article ๐Ÿ“… 1991 ๐Ÿ› Elsevier Science ๐ŸŒ English โš– 741 KB

Patients with severe combined immunodeficiency disease represent a model for the first clinical applications of gene therapy. Present attempts use insertion of the human adenosine deaminase gene into the peripheral blood T lymphocytes of patients who lack this gene. The ultimate treatment, however,