Abstract
San Raffaele Telethon Institute for Gene Therapy is developing an adenosine deaminase-transduced hematopoietic stem cell therapy for the potential intravenous treatment of adenosine deaminase deficiency in severe combined immunocompromised individuals.
Publication types
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Research Support, Non-U.S. Gov't
MeSH terms
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Adenosine Deaminase / deficiency
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Adenosine Deaminase / genetics
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Adenosine Deaminase / metabolism*
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Animals
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Cells, Cultured
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Clinical Trials as Topic
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Drug Evaluation, Preclinical
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Gene Transfer Techniques
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Genetic Therapy / methods
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Hematopoietic Stem Cell Transplantation* / methods
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Hematopoietic Stem Cells / metabolism*
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Humans
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Severe Combined Immunodeficiency / enzymology
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Severe Combined Immunodeficiency / genetics
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Severe Combined Immunodeficiency / therapy*
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Transplantation Conditioning*