Gene therapy for immunodeficiency |
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Authors: | Fabio Candotti MD |
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Affiliation: | (1) Clinical Gene Therapy Branch, National Human Genome Research Institute, National Institutes of Health, 10 Center Drive, Building 10, Room 10C103, 20892-1851 Bethesda, MD |
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Abstract: | Since the early 1990s, primary immunodeficiency (ID) disorders have played a major role in the development of human gene therapy.
Adenosine deaminase (ADA) deficiency was the first disease to be treated with a gene therapy approach in humans, and was also
the first condition for which therapeutic gene transfer into the hematopoietic stem cell has been attempted in the clinical
arena. A series of encouraging results obtained in chronic granulomatous disease (CGD) pateints have followed these pioneer
experiments and preceded the very recent and exciting reports of successful genetic correction procedures performed in patients
affected with the X-linked form of severe combined immunodeficiency (XSCID). The technical progress made in the field of gene
transfer in recent years is mostly responsible for these clinical advances, and will be critical for future development of
gene therapy approaches for other forms of IDs. |
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Keywords: | |
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