Article

Molecular mechanisms of immune deficiency in adenosine deaminase (ADA)-deficient SCID patients : implications for stem cell gene therapy

2007-01-01

Abstract excerpt

Mutations in the Adenosine Deaminase (ADA) gene are responsible for a form of Severe Combined Immunodeficiency (SCID), explained by the lymphotoxic accumulation of ADA substrates, adenosine and 2'-deoxy-adenosine. Haematopoietic stem cells (HSCs)-based gene therapy (GT) has been shown to correct both the immune and metabolic defect of ADA-SCID children, thus providing a unique model to investigate the molecular me...

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Literature Corpus work: f6dcc4de-9ab4-57ee-9e22-47d561667496
DOI: 10.21954/ou.ro.0000fa26

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