Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency
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Open Access
- 29 January 2009
- journal article
- research article
- Published by Massachusetts Medical Society in New England Journal of Medicine
- Vol. 360 (5), 447-458
- https://doi.org/10.1056/nejmoa0805817
Abstract
We investigated the long-term outcome of gene therapy for severe combined immunodeficiency (SCID) due to the lack of adenosine deaminase (ADA), a fatal disorder of purine metabolism and immunodeficiency.Keywords
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