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- Title
An Emerging Era of Clinical Benefit From Gene Therapy.
- Authors
Malech, Harry L.; Ochs, Hans D.
- Abstract
The article discusses gene therapy in patients with severe phenotype Wiskott-Aldrich syndrome (WAS), focusing on the restoration of immune function. Topics include opportunistic viral infections and autoimmune complications; the therapy of myeloid/lymphoid conditioning with an infusion of lentivector-transduced autologous CD34+ hematopoietic stem cells (HSCs); and correction of immune deficiencies, thalassemia, and metabolic disorders.
- Subjects
WISKOTT-Aldrich syndrome; GENE therapy; HEMATOPOIETIC stem cells; IMMUNE response; IMMUNOLOGICAL deficiency syndromes; THALASSEMIA treatment; VIRAL disease prevention; THERAPEUTICS
- Publication
JAMA: Journal of the American Medical Association, 2015, Vol 313, Issue 15, p1522
- ISSN
0098-7484
- Publication type
Opinion
- DOI
10.1001/jama.2015.2055