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- Title
Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement.
- Authors
Aiuti, Alessandro; Vai, Sergio; Mortellaro, Alessandra; Casorati, Giulia; Ficara, Francesca; Andolfi, Grazia; Ferrari, Giuliana; Tabucchi, Antonella; Carlucci, Filippo; Ochs, Hans D.; Notarangelo, Luigi D.; Roncarolo, Maria Grazia; Bordignon, Claudio
- Abstract
Presents letters to the editor on immune reconstitution in adenosine deaminase -severe combined immunodeficiency (ADA-SCID) after peripheral blood lymphocytes gene therapy and discontinuation of enzyme replacement. Difficulty in the assessment of clinical impact; Impact of polyethylene glycol on the defective cell population in ADA-SCID; Growth in the number of gene transduced T lymphocytes after the discontinuation of therapy.
- Subjects
ADENOSINE deaminase; ENZYME regulation; IMMUNE system
- Publication
Nature Medicine, 2002, Vol 8, Issue 5, p423
- ISSN
1078-8956
- Publication type
Letter
- DOI
10.1038/nm0502-423